Pharmaceutical Technology Is Now PharmTech: A Sharper Name for a Smarter Industry

Navigating FDA Shakeups and Obesity Drug Wars

Behind the Headlines Episode 28

Ask the Expert

Outsourcing Partnerships Fuel Global Biopharma Discovery and Scale-Up

Drug Digest February 2026 

How It Helps Avoid Facility and Equipment Problems

A “Plan, Prevent, Prove” Approach

Head of Radiopharmacology, Champion Oncology

Denis Beckford Vera, head of Radiopharmacology, Champion Oncology explains how patient-derived xenografts improve drug biodistribution studies and first-in-human dose accuracy.

Why PDX Models Improve Radiopharmaceutical Biodistribution Studies

Denis Beckford Vera, Head of Radiopharmacology, Champion Oncology, makes a case for incorporating patient-derived xenografts (PDXs) into the early stages of radiopharmaceutical development. He argues that doing so leads to more accurate, clinically relevant results and fewer costly setbacks down the line.

At the core of Vera's argument is the value of biodistribution studies conducted in PDX models rather than traditional immortalized cell lines. PDX tumors more faithfully replicate the architecture, vascularity, and receptor density of real patient tumors. They give researchers a far truer picture of how a radiopharmaceutical will actually behave in the body. Vera explains, "If you start early in the development using these patient-derived xenografts to evaluate the biodistribution of your radiopharmaceutical, even to choose lead compounds, you're going to have the real assessment of how your radiopharmaceutical is behaving."

One of the distinctive advantages of radiopharmaceuticals, is their inherent visibility. Researchers can directly observe where a compound travels and how it accumulates, making biodistribution an especially powerful tool for optimization. This real-time insight can streamline the path from preclinical research to first-in-human trials.

A major concern he raises is the tendency to overestimate first-in-human doses when relying on conventional cell line models, which often exhibit artificially high and homogeneous receptor expression. PDX models, by contrast, capture the heterogeneity seen across and within patients. Vera explains, "If you, from the beginning, use clinically relevant, preclinical models like patient-derived xenografts, you will have a real understanding of how much this drug is going to the tumor, and then that will directly impact the first-in-human dose estimation."

Ultimately, Vera's message is one of efficiency and precision, building on clinically relevant models from the outset reduces the likelihood of having to return for costly re-optimization later in development.

Editor's note: This transcript is a lightly edited rendering of the original audio/video content. It may contain errors, informal language, or omissions as spoken in the original recording.

I'm Denis Beckford Vera. I am the head of radiopharmacology at Champion Oncology. This is very related to clinical translation. So if you start early in the development using these patient-derived xenografts to evaluate the biodistribution of your radiopharmaceutical, even to choose lead compounds, you're going to have the real assessment of how your radiopharmaceutical is behaving. Moving from biodistribution, I think biodistribution should be the first type of assessment that you do preclinically in these, clinically relevant models, to understand where target engagement, for instance, in the tumor. How this tumor heterogeneity, and how this, tumor architecture, that mimics the patient's tumor, can really affect the biodistribution of the radiopharmaceutical, which is very tied to vascularity, for example. It is different in immortalized cell lines than in real patient samples. The receptor density and how the receptors distribute between the tumor and between, different patients and the lesions between the same patient as well. So you're going to get an idea of how this can really distribute. More importantly, you can really save time because with radiopharmaceuticals, you have something that you don't have with other modalities. You can see, you can see your real molecule. So if you are able to see where it goes and how it behave where it goes. So then you can optimize the process for later efficacy studies in this same tumor model. Even further, if there is a biologically relevant species, you can also identify off-target toxicity as well doing biodistribution studies, which is very important, very relevant to, to these types of treatments. From the beginning, you are evaluating your compound in clinically relevant models. You later don't have to come back and do optimization, what happens many times. So this is what happens, when you use biodistribution to assess a first-in-human studies and to estimate first-in-human doses. So many times, this dose is overestimated, based on these regular cell line-derived xenografts. We have high receptor expression, in many cases, has more homogeneous receptor expression. And then you're saying, you are getting a wrong understanding on how much your drug is really accumulating in those tumors. So if you, from the beginning, use clinically relevant, preclinical models like patient-derived xenografts, you will have a real understanding of how much this drug is going to the tumor, and then that will directly impact the first-in-human dose estimation. So you will not have to go back and do, or you will have the probability that you will have to go back and do so much optimization to improve in human doses and things like that, will be less likely, than if you went right.

Videos

FDA Approves J&J’s Icotrokinra | Created with Canva

), an interleukin-23 receptor antagonist for the treatment of moderate-to-severe plaque psoriasis.¹ 

This treatment, developed by Johnson & Johnson

 in collaboration with Protagonist Therapeutics, is designed for adults and pediatric patients 12 years and older weighing at least 40 kg who are candidates for systemic therapy or phototherapy.

The approval marks a meaningful shift in the oral small molecule and peptide therapeutic landscape.¹ Unlike existing oral agents, icotrokinra is a targeted oral peptide, which has historically faced significant bioavailability and selectivity challenges in development. Its successful path through Phase III represents a proof of concept that peptide-based receptor antagonism can be delivered orally at therapeutic levels, a finding relevant to pipeline programs exploring similar modalities.

The drug works by binding to the IL-23 receptor with high affinity, inhibiting downstream signaling in human T cells.¹ The clinical significance of receptor occupancy data is noted as unknown, but the mechanism positions icotrokinra at the same biological target as several approved injectable biologics.

What Does the Clinical Evidence Base Look Like?

The approval rests on the Iconic program, a Phase III development package spanning five studies and approximately 2,500 patients.¹ This dataset is notable for its scope and for the simultaneous enrollment of adult and adolescent populations, which often requires separate development tracks.

The package included duplicate head-to-head superiority trials (Iconic-Advance 1 and 2) against deucravacitinib, an approved oral tyrosine kinase 2 inhibitor.¹ Across those trials, roughly 70% of patients achieved an Investigator's Global Assessment score of 0 or 1 and approximately 55% reached a 90% improvement in Psoriasis Area and Severity Index score at Week 16. Running head-to-head trials in duplicate is an uncommon regulatory strategy and reflects a deliberate effort to generate robust comparative evidence.

Safety data through Week 16 showed adverse reaction rates within 1.1 percentage points of placebo, with no new safety signals identified through Week 52.¹ The most common adverse reactions were headache, nausea, cough, fungal infection, and fatigue. From a benefit-risk modeling perspective, the clean short-term profile, combined with a once-daily oral regimen taken on an empty stomach, presents a favorable adherence scenario compared with injectable alternatives.

"With new guidance from the International Psoriasis Council that clarifies when to move beyond cycling on topical treatments to systemic therapy, an innovative option like ICOTYDE is a potential game-changer for many adult and adolescent patients," said Linda Stein Gold, MD, director of Dermatology Clinical Research, Henry Ford Health, in a press release.¹

What Are the Downstream Implications for the Pipeline?

Johnson & Johnson retains exclusive worldwide development and commercialization rights under its license and collaboration agreement with Protagonist.¹ Icotrokinra is now in active investigation across additional indications (psoriatic arthritis (two studies), ulcerative colitis, and Crohn's disease) meaning the manufacturing and regulatory infrastructure built for this approval will need to scale accordingly.

The oral peptide format raises specific process considerations around stability, formulation, and GMP production that differ from both small molecules and conventional biologics.¹ The fact that the molecule can be dispersed in water and is dosed on an empty stomach suggests formulation constraints that likely shaped the development and manufacturing strategy.

"At Johnson & Johnson, we are harnessing our scientific expertise to transform cutting-edge science into meaningful solutions for patients," said John Reed, MD, PhD, executive vice president, R&D, Innovative Medicine, Johnson & Johnson, in the press release.¹ "ICOTYDE is a fundamentally different treatment with the potential to redefine what physicians and patients can expect from psoriasis treatment. The approval of ICOTYDE represents a pivotal moment for people with plaque psoriasis.”

How far Does the Oral Peptide Precedent Extend Beyond Psoriasis?

The icotrokinra approval arrives alongside broader momentum in Johnson & Johnson's targeted therapy pipeline.² At the European Association of Urology's 2026 Annual Meeting, the company presented Phase I results for erdafitinib intravesical drug-releasing system (Erda-iDRS), an investigational intravesical delivery system designed to release the kinase inhibitor erdafitinib directly into the bladder over three months, which minimizes systemic exposure while sustaining localized drug levels.

In 62 patients with recurrent intermediate-risk non-muscle-invasive bladder cancer harboring fibroblast growth factor receptor alterations, the complete response rate reached 89%, with a median response duration of 18 months.²

"The high and durable complete responses demonstrated with Erda-iDRS highlight the opportunity to deliver a targeted therapy to these patients," said Christopher Cutie, M.D., Vice President, Disease Area Leader, Bladder Cancer, Johnson & Johnson, in a press release.² Phase III studies are ongoing.

Johnson & Johnson. FDA approval of ICOTYDE (icotrokinra) ushers in new era for first-line systemic treatment of plaque psoriasis with a targeted oral peptide. News release. March 17, 2026. Accessed March 18, 2026. 

https://www.jnj.com/media-center/press-releases/fda-approval-of-icotyde-icotrokinra-ushers-in-new-era-for-first-line-systemic-treatment-of-plaque-psoriasis-with-a-targeted-oral-peptide

​Johnson & Johnson. Johnson & Johnson highlights promising first-in-human ERDA-IDRS (formerly TAR-210) results in intermediate-risk non–muscle-invasive bladder cancer. News release. March 18, 2026. Accessed March 18, 2026. 

https://www.jnj.com/media-center/press-releases/johnson-johnson-highlights-promising-first-in-human-erda-idrs-formerly-tar-210-results-in-intermediate-risk-non-muscle-invasive-bladder-cancer

Articles

FDA approval of icotrokinra, an oral IL‑23 receptor–blocking peptide, expands systemic options for moderate-to-severe plaque psoriasis in patients ≥12 years and ≥40 kg. The Phase III Iconic program (~2,500 participants) included duplicate superiority head-to-head trials versus deucravacitinib, yielding Week‑16 IGA 0/1 rates near 70% and PASI90 around 55%. Adverse events were comparable to placebo at Week 16 and remained without new signals through Week 52. Commercialization now supports expansion into psoriatic arthritis and IBD; J&J also advanced intravesical erdafitinib with 89% complete responses in FGFR-altered NMIBC.

The FDA approves icotrokinra, the first targeted oral peptide blocking the IL-23 receptor, for moderate-to-severe plaque psoriasis with Phase III data across 2,500 patients.

FDA Approves First Oral Peptide for Plaque Psoriasis

Environmental technology concept. Sustainable development goals. SDGs.

| Image Credit: ©metamorworks - stock.adobe.com

Pharmaceutical companies are forging sustainability initiatives that include investments in renewables, refurbishing facilities to net zero, and committing to a greener industry.

In addition, regulators and industry organizations such as the World Health Organization (WHO) are calling for more sustainable manufacturing practices in the pharma industry.

“Addressing the environmental impact of healthcare products is no longer optional—it is imperative,” said Yukiko Nakatani, MD, PhD, assistant director-general for access to medicines and health products at WHO. “The transformation of regulatory practices will be pivotal in shaping a pharmaceutical industry that meets today’s health needs without compromising the planet’s future.”

As the shift to sustainability becomes a core metric of success alongside speed and quality, companies are locking in environmental efficiencies during the early development phase, ensuring that program move to manufacturing with the smallest possible footprint.

 spoke with Andrew Moreo, head of process development and operations management at Andelyn Biosciences; Rahul Mittal, head of strategy & innovation at Dr. Reddy’s Laboratories; and Andrew Whitley, PhD, vice-president of global business development, life science field officer, at HORIBA; David Butler, chief technology officer, Hongene; and SreeKrishna Chopperla, senior vice president, HSE, Sai Life Sciences, about the strategies pharma companies are creating to move toward more sustainable manufacturing.

What specific changes has your company made in your drug manufacturing practices to improve sustainability?

Sustainability in manufacturing is not an afterthought; it is a design choice made at the bench. At Andelyn, we recognize that the most significant environmental impact of a drug is locked in during the development phase. To address this, we utilize optimization-by-design. This methodology uses advanced data analytics and design of experiments to identify the most efficient process parameters before we ever step into a clean room. By refining the process early in process development, we ensure that when a program moves to good manufacturing practice, it does so with the smallest possible footprint of raw materials and energy.

Dr. Reddy’s has made targeted, measurable changes in its manufacturing practices to strengthen sustainability. The company is transitioning its sites to 100% renewable power by 2030 and has already reached 60% progress toward carbon‑neutral Scope 1 and 2 operations. It has also become a water‑positive manufacturer, consistently replenishing more water than it consumes. Waste systems have been redesigned so that 99% of hazardous waste is co‑processed or recycled, significantly reducing landfill dependence. Cold chain packaging has been optimized, with over 90% of cold boxes reused across distribution points. These efforts align with global best practices, and the company’s recent earnings updates indicate that energy efficiency and operational discipline also support stronger margins.

Our focus has been on systematically redesigning processes using green chemistry principles and technology-driven innovation to minimize hazardous inputs, optimize solvent use, and improve water and energy efficiency. Across programs, we have strengthened solvent circularity, enhanced mass efficiency, and reduced waste through process innovation supported by digital monitoring. In parallel, we are deploying advanced manufacturing and green engineering solutions to structurally embed resource efficiency into our manufacturing platforms.

 We’ve focused on two levers—process design and site operations— to improve stability. On the process side, we’re advancing chemoenzymatic ligation for oligonucleotides to improve yield and cut solvent and purification burden versus traditional solid phase synthesis. On the operations side, we’ve added renewable energy elements (e.g., solar-powered exterior lighting), electrified on-site logistics (electric vehicles), and invested in solvent recycling. We also track progress through third-party ESG assessment (EcoVadis).

Which area/process of drug manufacturing impacts sustainability efforts the most?

The largest sustainability impact comes from API and chemical‑synthesis operations, which drive the bulk of energy consumption and emissions. This is why manufacturers prioritize decarbonization and report steady progress toward carbon‑neutral operations.

These steps also require significant water for purification and cleaning, making water‑stewardship programs essential, including recycling loops that support water‑positive outcomes. Hazardous waste generation is another major factor; companies now report over 99% diversion of such waste to co‑processing and recycling at scale. In contrast, packaging and logistics have lower environmental intensity, though cold chain reuse helps reduce secondary waste.

The greatest sustainability impact is determined at the process design stage. Reaction pathways and downstream isolation steps—particularly crystallization and filtration—largely define solvent intensity, energy consumption, water usage, and waste generation. When these elements are optimized early, companies can apply green chemistry principles and technology-enabled process enhancements to significantly reduce environmental burden and operational variability. In our view, sustainability is fundamentally a chemistry and engineering decision made at the core of process development.

 In nucleic acid manufacturing, sustainability is often assessed using process mass intensity (PMI), which is mainly driven by solvent and reagent consumption in synthesis and purification. Traditional solid-phase oligonucleotide synthesis is highly solvent-intensive and generates substantial chemical waste which drives PMI higher. We’re prioritizing next-generation technologies that employ biocatalysis to improve yields and reduce PMI and energy consumption while also lowering cost.

In viral vector manufacturing, inefficiency is the primary driver of waste. The area where we can create the greatest impact is within the underlying power of a manufacturing platform, which is why we focus so heavily on our AAV Curator Platform. This allows us to leverage standardization of materials, equipment, and systems, with flexibility to enable us to achieve higher yields and better full-to-empty capsid ratios from the start. By significantly increasing the usable product per batch, we effectively reduce the number of manufacturing runs required to meet the clinical goal. In the world of advanced therapies, yield is the ultimate sustainability lever because higher productivity directly translates to fewer consumables and a smaller carbon footprint per dose.

How can pharma manufacturers improve their manufacturing process to ensure better sustainability?

 As I have already emphasizsed improvement begins with designing processes differently—not merely operating them more efficiently. This involves prioritizing green chemistry, improved atom economy, circular resource use, and technology-enabled optimization from the outset. Transitioning from traditional batch manufacturing to continuous processing, and adopting process intensification and green manufacturing technologies, can significantly reduce variability, waste, and energy consumption. Most importantly, sustainability must be embedded during early development, where decisions have the greatest long-term environmental and economic impact.

A major opportunity for improvement lies in harmonizing global regulations. Having a single source of regulatory standards across regions would enable smoother logistical transitions for vendor-supplied materials, manufacturing, and final product shipment. When requirements are aligned globally, manufacturers can avoid redundant systems, duplicate testing, and unnecessary supply chain workarounds that add cost, complexity, and waste.

At the same time, facility design plays a critical role. Purpose-built manufacturing facilities, designed with efficiency from the outset, offer a significant sustainability advantage over retrofitted sites. This is why many organizations find that partnering with a CDMO [contract development and manufacturing organization] is a more sustainable path than building or managing in-house production in less optimized environments. An intentionally built-for-purpose facility allows manufacturers to right-size utilities, optimize material flows, and incorporate energy-efficient systems to reduce waste throughout the facility’s life cycle. Sustainability is not just about scale but about intentional design decisions that enable large, complex operations to run more efficiently and consistently.

 Manufacturers can improve sustainability by expanding renewable‑energy sourcing and carbon‑neutral direct operations. Demand forecasting methodologies help in batch processing. AI tools are assisting companies with a shorter path to drug development and reducing wastage of chemical solvents [and] intermediaries and saving electricity.

Water efficiency must improve through advanced recycling, membrane filtration, and real‑time monitoring so plants achieve water‑positive performance. Waste reduction requires stronger solvent‑recovery systems and partnerships that route nearly all hazardous waste to co‑processing, a benchmark some companies already meet. Digital tools such as predictive maintenance and automated analytics improve yield and reduce batch failures, contributing to better margins.Reusable cold chain packaging and lighter materials further reduce environmental load. Sustainability and efficiency become mutually reinforcing when these measures scale.

Replacing or complementing chromatography with optical spectroscopy will allow for less reliance on solvents. Spectroscopy generally does not require solvents, and therefore, there are no chemical waste products. The added upside here is that this also means significantly less cost of ownership. Everybody wins. As spectroscopy is typically much faster than chromatography and uses less energy, there is also a significant reduction in power required to obtain the required information.

 It’s important to measure consistently and set clear targets. Track PMI, energy usage, waste recycling rates, and Scope 1–3 emissions, then prioritize work based on the biggest drivers. Practical steps include reducing solvent-intensive unit operations, adding solvent recovery, and choosing higher-yield routes that use biocatalysis to reduce rework and purification burden. Stronger quality systems can also help by reducing deviations and repeat batches. Finally, simplify supply chains where possible because fewer handoffs and less transportation generally mean a lower footprint and better control.

How do you see sustainability impacting the industry in the future?

In many European countries, a reduction in carbon footprint, minimal waste, and environmentally safe elimination of any waste are already mandated and regulated. The push for improvements will only increase. Meeting these sustainability requirements will be challenging and could be very expensive. The use of optical spectroscopy, as discussed, will help pharma support these requirements.

 Our interactions with pharma indicate that sustainability is increasingly a partner-selection criterion, not just a reporting topic. Developers are asking for quantified metrics such as PMI, emissions, and waste, and they expect credible improvement plans aligned with recognized standards. Over time, processes and technologies will shift toward platforms that scale with lower PMI and stronger supply reliability. We believe the winners will be manufacturers that can prove sustainability while maintaining or improving supply reliability and cost.

There is no one-size-fits-all solution to the sustainability challenges of drug manufacturing. Instead, we must approach this challenge from all sides simultaneously by integrating better science through optimization-by-design, more efficient platforms, and smarter facility management. As the industry matures, sustainability will become a core metric of success, alongside speed and quality. We will not find a single silver bullet for sustainability. Rather, we will find success through the silver buck-shot of a thousand small, intentional efficiencies across the entire manufacturing lifecycle.

Sustainability, while being critical in conversations right now, is expected to be a core competitive requirement and be featured within strategy discussions and earnings reports.

Companies already commit to carbon neutrality, full renewable‑energy transitions, and water‑positive operations, signaling that environmental performance will shape investment, procurement, and regulatory expectations.

Annual sustainability disclosures supported by independent assurance will become standard, reinforcing transparency. Operational efficiency and sustainability will converge, since earnings commentary increasingly connects energy management and manufacturing discipline with improved margins. Supply chains will be redesigned around resilience, renewable inputs, and lower Scope 3 emissions. Regulations will tighten, and customers will prefer manufacturers with verifiable sustainability records. Over time, sustainability will define how medicines are made, how risks are managed, and how companies differentiate in global markets.

Sustainability is evolving into both a strategic differentiator and an industry baseline. Customers are increasingly evaluating environmental performance alongside cost and timelines, including process efficiency, resource circularity, and alignment with green chemistry principles. In the coming years, manufacturing platforms will be designed for resilience, efficiency, and a reduced environmental footprint from the ground up. Organizations that integrate green chemistry with resource-conscious engineering, supported by innovation and digitalization, will be best positioned to meet emerging regulatory expectations and market demand.

Sustainability in the pharmaceutical industry. Association of the British Pharmaceutical Industry

 Accessed February 17, 2026. https://www.abpi.org.uk/reputation/sustainability-in-the-pharmaceutical-industry/

WHO calls for transformative action towards a greener future in pharmaceutical manufacturing and distribution. World Health Organization. December 23, 2024. Accessed February 17, 2026https://www.who.int/news/item/23-12-2024-who-calls-for-transformative-action-towards-a-greener-future-in-pharmaceutical-manufacturing-and-distribution

Sustainable pharmaceutical manufacturing is increasingly driven by regulatory pressure, WHO advocacy, and corporate commitments to renewables, net-zero facilities, and quantified ESG performance. The largest environmental burdens are frequently fixed during early process and platform design, making optimization-by-design, green chemistry, and yield improvements critical levers. High-impact hotspots include API synthesis, solvent-intensive purification, and hazardous-waste generation; leading programs emphasize solvent circularity, water-positive operations, and near-total hazardous-waste diversion. Emerging enablers include biocatalysis, continuous processing, digital monitoring, and solvent-free analytical approaches.

Industry experts explain how incorporating sustainability into the early development phase creates greener pharmaceutical manufacturing.

Creating Sustainable Operational Excellence in Pharma Manufacturing

SGD Pharma’s Sterile Nasal Vial Platform | Created with Canva

On March 17, 2026, SGD Pharma announced a strategic original equipment manufacturer collaboration with Prince Sterilization Services, LLC to validate ready-to-use type I glass primary packaging solutions for the North American market.¹ The first commercial outcome of that collaboration is the My Sterinity Nasal platform, a flexible cleaned vial range built on SGD Pharma's existing My Sterinity platform that will be formally introduced at DCAT Week 2026 in New York.

The announcement signals the availability of a regionally qualified, end-to-end supply chain for sterile Type I glass nasal vials, an offering that has historically been difficult to source with short lead times in the US.¹

Why Is the Nasal Delivery Market Drawing Renewed Attention to Glass Packaging?

The global nasal drug delivery market is valued at approximately €9 billion ($10.4 billion) and encompasses more than 2 billion units annually.¹ Plastic has historically dominated this segment, accounting for roughly 88% of packaging formats. However, the therapeutic profile of nasal drug programs is shifting. What was once a market concentrated on acute and rescue treatments is now expanding into chronic, high-value indications for conditions that include Alzheimer's disease, Parkinson's disease, multiple sclerosis and depression.

These longer-duration therapies place more demanding requirements on primary packaging.¹ Formulation teams need containers that offer strong chemical inertness, consistent barrier protection and long-term biocompatibility. Minimizing adsorption of active compounds, preserving sterility over extended treatment courses and enabling precision dosing are increasingly non-negotiable for this category. Type I borosilicate glass satisfies these criteria in ways that plastic alternatives often cannot.

What Will the SGD Pharma and Prince Collaboration Deliver?

My Sterinity Nasal launches initially in three vial formats--0.5 mL, 7.5 mL and 10 mL--that are available in both clear and amber glass.¹ The vials are based on SGD Pharma's U-Save type I glass and offer controlled particulate and endotoxin levels, validated sterility assurance and compatibility with standard preservative-free nasal pump systems. They are packaged in double-bagged trays for immediate deployment in filling operations.

The division of responsibilities in the collaboration is designed to address a practical gap in the North American supply landscape.¹ Glass manufacturing will take place at SGD Pharma's Saint Quentin La-Motte facility in northern France, which holds ISO 15378 certification and operates to GMP standards. Prince will then handle washing, depyrogenation and sterilization services on the North American side, creating a qualified, regional supply chain suited to US biopharmaceutical customers.

Carole Grassi, chief commercial marketing & innovation officer, SGD Pharma, said in a press release,¹ “Our collaboration with Prince marks an important milestone in SGD Pharma's innovation growth strategy for North America. The My Sterinity Nasal platform represents a major step forward in high-value nasal drug delivery, and our collaboration with Prince unlocks immediate US-qualified capacity for biopharma customers scaling complex nasal therapy programs. This is more than a product launch, it is a validated supply chain ready to support qualification, clinical trials and commercial launch with unmatched speed and reliability.”

Daniel Prince, PhD, CEO, Prince Sterilization Services, said, in the press release,¹ "SGD Pharma's Type I glass expertise combined with our quality focused and flexible RTU & sterilization platform creates the gold standard for sterile nasal packaging. Prince's recent US qualification means we can now deliver ready-to-use, terminally sterilized My Sterinity Nasal vials in timelines that match domestic and international customer development cycles and exceed quality requirements."

The practical implication is access to terminally sterilized, type I molded glass vials without the delays typically associated with custom qualification projects or international shipping timelines.¹ SGD Pharma's broader type I molded glass portfolio is characterized by high hydrolytic resistance, low thermal expansion. This being relevant for lyophilization and autoclaving processes.

What Else Is Being Presented at DCAT Week 2026?

Alongside the My Sterinity Nasal launch, SGD Pharma will present two additional glass packaging technologies at DCAT Week.¹ 

Sealian is an internal surface treatment designed to improve chemical durability and barrier performance for sensitive drug formulations

. The company will also present Idency premium vials, which are engineered to provide greater chemical and mechanical resistance than standard tubular vials while retaining the same outer diameter. Idency allows integration with existing filling line configurations without equipment modification.

SGD Pharma will not be alone in addressing supply chain resilience in injectable and specialty drug segments.² Avenacy, a US-based specialty pharmaceutical company, will discuss its portfolio of twenty-six critical acute care and shortage injectable medications. The company is using the conference to engage potential customers and partners around further product launches aimed at stabilising supply in high-demand categories.

Lotte Biologics will also be present at DCAT Week 2026, using the event to advance CDMO partnership discussions in the North American market and beyond.³ The company plans to highlight the combined capabilities of its Syracuse bio campus in New York and Plant 1 at its Songdo facility in Incheon, South Korea. Lotte will present a cell line development to drug substance production timeline of approximately 8.5 months, achieved through a partnership with Asimov. Manufacturing support spans monoclonal antibodies, bispecific antibodies and both clinical and commercial-scale production. The presences of these organizations at DCAT Week 2026 reflects a broader industry preoccupation with qualified, reliable supply chains for complex drug delivery formats.

SGD Pharma. SGD Pharma advances its North American pharmaceutical glass offering in new collaboration with Prince Sterilization Services, LLC – debuting its My Sterinity Nasal range at DCAT Week 2026. Paris La Défense, France: SGD Pharma; March 17, 2026. Press release.

​Avenacy. Avenacy to attend 2026 DCAT Week. Business Wire. March 16, 2026. Accessed March 18, 2026. 

https://www.businesswire.com/news/home/20260316315025/en/Avenacy-to-Attend-2026-DCAT-Week

Korea Biomedical Review. Lotte Biologics to participate in DCAT Week 2026 in New York. Korea Biomedical Review. March 17, 2026. Accessed March 18, 2026. 

https://www.koreabiomed.com/news/articleView.html?idxno=30848

SGD Pharma and Prince Sterilization Services have established an OEM collaboration to validate ready-to-use Type I glass primary packaging for North America, debuting the My Sterinity Nasal platform at DCAT Week 2026. The initiative addresses persistent US sourcing constraints for sterile nasal vials by creating a regionally qualified, end-to-end supply chain. Momentum reflects a nasal delivery market shifting from acute use toward chronic, high-value CNS and immunologic indications, increasing the need for inertness, barrier performance, sterility retention, and dosing precision.

SGD Pharma and Prince launch sterile Type I glass nasal vials for the US market, targeting complex chronic conditions like Alzheimer's and Parkinson's.

SGD Pharma Launches Sterile Nasal Vial Platform for US Market

Med Uni Graz and Boehringer Lung Cancer Research Lab | Created with Canva

The Medical University of Graz and Boehringer Ingelheim have opened a Christian Doppler Laboratory dedicated to developing new therapies for non-small cell lung cancer, with a specific focus on immunogenic cell death.¹ The 7-year program, funded at €3.2 million (US$3.7 million) split equally between Boehringer Ingelheim and public funding bodies, represents a structured attempt to close one of oncology's most persistent gaps, durable response rates in a disease that remains the leading cause of cancer-related deaths worldwide.

The research agenda here is notable for its specificity.¹ Rather than pursuing broad immunotherapy mechanisms, the laboratory is targeting the upstream biology that determines whether a tumor is visible to the immune system at all.

Why Does Immunogenic Cell Death Matter for Drug Development?

Cells in the human body have a natural self-destruct program.¹ When damaged or no longer needed, they die in a controlled manner a process essential to maintaining the body's health. In certain cases, known as immunogenic cell death, dying cells emit signals that actively recruit immune cells to potential threats. In non-small cell lung cancer, these mechanisms are frequently suppressed, allowing tumors to proliferate undetected.

"In non-small cell lung cancer, these mechanisms are often switched off. As a result, the tumor can grow undetected by the immune system,"¹ explained Philipp Jost, director of the Division of Oncology, Medical University of Graz, in a press release.

"The aim of our research is therefore to influence cancer cells in such a way that they can be more easily recognized and attacked by the immune system. If successful, existing cancer therapies could become significantly more effective,”¹ noted Michael Dengler, co-lead, Christian Doppler Laboratory, in the press release.

The hypothesis is not that a new molecule replaces existing therapies, but that restoring immunogenic signaling could meaningfully extend the utility of treatments already in clinical use, including targeted therapies and immunotherapies that currently offer only temporary benefit before relapse.¹

What Does This Mean for Translational Pipeline Strategy?

The laboratory's structure is designed specifically to accelerate the journey from mechanistic insight to clinical application.¹ Located on the Medical University of Graz campus within Medical Science City Graz, the lab has access to clinically relevant samples through Biobank Graz, providing the kind of human-derived data that often proves the critical bottleneck in early translational work.

Boehringer Ingelheim, which coordinates its global oncology research from Austria through its Regional Center Vienna, a site focused on cancer biology, computational innovation, and translational research, brings industry-side development expertise to the collaboration.¹ "This partnership brings together complementary strengths: the scientific excellence and clinical integration of Med Uni Graz and our expertise in the discovery and development of new cancer therapies. Together, we aim to accelerate the development of treatments that have the potential to sustainably change the course of the disease,"¹ said Mark Paul Petronczki, head of Oncology Research, Boehringer Ingelheim, in the press release.

The laboratory's leads describe their core work as investigating the biological mechanisms that determine how and when lung cancer cells die.¹ This means the near-term outputs are likely to be mechanistic data and validated biological targets, rather than late-stage candidates. The program is structured to move findings toward clinical application with an industry partner already positioned to act on them.

Andrea Kurz, rector, Medical University of Graz, framed the intent, in the press release,¹ "Through our partnership with Boehringer Ingelheim, we are strengthening our ability to translate new scientific insights into transformative therapeutic approaches for patients. This Christian Doppler Laboratory provides an outstanding framework to connect basic research with clinical application and to accelerate innovation where it is needed most."

The Christian Doppler Research Association model is a well-established Austrian mechanism for this kind of public-private research collaboration. Federal Minister Wolfgang Hattmannsdorfer, noted, in the press release,¹ "This Christian Doppler Laboratory is a prime example of how successful innovation policy works: when science and industry collaborate closely, innovation, value creation, and high-quality jobs emerge."

What Are the Other Notable Developments in Med Uni Graz?

Separate research may carry implications for how the field approaches cell detection more broadly.² A team at the Medical University of Graz has published findings on microchimerism including the first successful application of a new technology for detecting extremely rare cells. "Now that we are able to detect these cells more directly, sensitively and reliably, new opportunities in research and diagnostics are opening up," said Thomas Kroneis, project leader of the Division of Cell Biology, Histology and Embryology at the Gottfried Schatz Research Center for Cell Signaling, Metabolism and Aging, Med Uni Graz, in a press release.² The methodology, developed in part through a $5.34 million John Templeton Foundation grant, may have downstream applications in cancer research and diagnostics.

Medical University of Graz. Med Uni Graz and Boehringer Ingelheim open new Christian Doppler Laboratory. Published March 17, 2026. Accessed March 17, 2026. 

https://www.medunigraz.at/en/news/detail/medizinische-universitaet-graz-und-boehringer-ingelheim-eroeffnen-neues-christian-doppler-labor

Medical University of Graz. Neue Einblicke in den Mikrochimärismus vorgestellt. Published March 4, 2026. Accessed March 17, 2026. 

https://www.medunigraz.at/en/news/detail/neue-einblicke-in-den-mikrochimaerismus-grazer-forschung-setzt-internationale-massstaebe

A new Christian Doppler Laboratory at the Medical University of Graz, partnered with Boehringer Ingelheim, will run a 7-year, €3.2 million program to enhance non-small cell lung cancer therapy by reactivating immunogenic cell death pathways. By restoring danger signaling that promotes immune recognition, the effort aims to boost durability of current targeted agents and immunotherapies. Embedded in Medical Science City Graz with access to Biobank Graz and industry development capabilities from Boehringer’s Vienna oncology hub, the program emphasizes mechanistic discovery and target validation for translation.

The new €3.2M (US$3.7M) research lab will investigate immunogenic cell death mechanisms in lung cancer to improve how existing therapies trigger immune response.

Med Uni Graz and Boehringer Ingelheim Open Lung Cancer Research Lab

FDA Accepts Tildrakizumab-asmn | Created with Canva

The FDA has accepted for review a supplemental Biologics License Application (sBLA) for Sun Pharma’s tildrakizumab-asmn to treat adults with active psoriatic arthritis.¹ This represents a significant step in the lifecycle management of a humanized monoclonal antibody designed to selectively bind to the p19 subunit of interleukin-23 (IL-23). By inhibiting the interaction with the IL-23 receptor, the treatment aims to prevent the release of pro-inflammatory cytokines and chemokines. The FDA action date for this regulatory decision is anticipated by October 29, 2026.

How Does this Impact the Development of Interleukin-23 Inhibitors?

Expanding the indications of an existing biologic is a critical strategy for addressing unmet needs in psoriatic disease, where approximately one in three patients with psoriasis eventually develops psoriatic arthritis.¹ Since its initial 2018 approval for moderate-to-severe plaque psoriasis, tildrakizumab-asmn has seen its label expand to include scalp and nail psoriasis in 2024 and 2025, respectively. Rick Ascroft, CEO, Sun Pharma North America, noted, in a press release,¹ “For many people living with psoriatic disease, joint symptoms often add another layer of burden.” These expansions highlight the importance of long-term clinical follow-up, which for this biologic currently includes 5 years of data showing a well-characterized safety profile.

What Clinical Evidence Underpins the Proposed Expansion for Psoriatic Arthritis?

The current sBLA submission is supported by data from the INSPIRE-1 and INSPIRE-2 Phase III clinical studies.¹ These 52-week global, multicenter, randomized, double-blind, placebo-controlled trials evaluated the efficacy and safety of a 100 mg dose in adult patients with active psoriatic arthritis. Regarding the strategic direction of the product, Ascroft stated, in the press release,¹ “As we continue to strengthen Sun Pharma’s innovative portfolio, we look forward to working with the FDA throughout the review process. As the only HCP-administered IL-23 biologic, our ambition is that ILUMYA becomes a differentiated first-choice advanced systemic treatment for active psoriatic arthritis.” The move toward a HCP administered option for psoriatic arthritis differentiates the product in a competitive market while leveraging a global infrastructure that has already secured marketing authorization from over 55 health authorities. From a safety perspective, common adverse reactions recorded in trials include upper respiratory infections, injection site reactions, and diarrhea. The review process will continue through 2026, building upon the treatment's existing history of supporting nearly 140,000 patients worldwide.

The manufacturer's specialty portfolio, which accounts for approximately 20% of global sales, is expanding beyond immunology into onco-dermatology.² A significant milestone in this expansion is the US availability of cosibelimab-ipdl for adults with metastatic or locally advanced cutaneous squamous cell carcinoma (aCSCC) who are not candidates for curative surgery or radiation. This agent represents a technical evolution in checkpoint inhibition as a programmed death-ligand 1 (PD-L1) antibody that preserves programmed death-ligand 2 (PD-L2) signaling. Ascroft stated, in a press release,² “UNLOXCYT is an evolution in checkpoint inhibition, combining durable efficacy with a proven tolerability profile for a group of aCSCC patients who traditionally would struggle to strike that therapeutic balance.”

How Is the Oncology-dermatology Portfolio Evolving with New Checkpoint Inhibitors?

The clinical utility of this PD-L1 therapy is evidenced by a 71% disease control rate in the CK-301-101 trial.² Ann W. Silk, MD, MS, medical oncologist, Dana-Farber Cancer Institute, explains, in the press release,² “Because these patients tend to be older and have multiple comorbidities, it’s extremely valuable to have a therapy that offers durable disease control and proven tolerability.” This matters to development teams because the multifaceted mechanism of action involves both adaptive and innate immune responses while maintaining PD-L2 signaling.

What Clinical Outcomes Define the Performance of this Novel Antibody?

The product requires a limited distribution network for its 1,200 mg intravenous infusion administered every three weeks.² While common reactions include fatigue and musculoskeletal pain, long-term data confirms durable efficacy and a proven tolerability profile. These advancements reinforce the manufacturer's established reach across six continents.

Sun Pharmaceutical Industries Limited. Sun Pharma announces US FDA acceptance of supplemental Biologics License Application for ILUMYA (tildrakizumab-asmn) for the treatment of adults with active psoriatic arthritis. News release. March 16, 2026.

Sun Pharmaceutical Industries Limited. Sun Pharma announces the availability of UNLOXCYT (cosibelimab-ipdl) for advanced cutaneous squamous cell carcinoma (aCSCC). News release. January 15, 2026. Accessed March 16, 2026. 

https://sunpharma.com/usa/wp-content/uploads/sites/2/2026/01/UNLOXCYT-Commercial-Launch-Press-Release.pdf

FDA accepted an sBLA for tildrakizumab-asmn in active psoriatic arthritis, with a PDUFA target of October 29, 2026. The IL‑23p19 monoclonal antibody, approved for plaque psoriasis since 2018 with recent scalp and nail label expansions, is supported by 52‑week INSPIRE-1/2 Phase III data at 100 mg. Sun Pharma positions it as the only HCP-administered IL‑23 biologic, citing five-year safety follow-up and ~140,000 treated patients. Concurrently, the company is expanding into onco-dermatology with cosibelimab-ipdl for advanced cSCC and a 71% disease control signal in CK-301-101.

The FDA will evaluate tildrakizumab-asmn for psoriatic arthritis. A 2026 decision is expected based on Phase III clinical trial data.

FDA Accepts Tildrakizumab-asmn sBLA for Active Psoriatic Arthritis

Denis Beckford Vera, head of Radiopharmacology, Champion Oncology, discusses PDX models preserving tumor architecture and heterogeneity, yielding clinically relevant data for more accurate radiopharmaceutical translation.

Improving Clinical Translation of Radiopharmaceuticals Using PDX Models

Denis Beckford Vera, head of Radiopharmacology, Champion Oncology, discusses the critical role of patient-derived xenograft (PDX) models in advancing radiopharmaceutical development. PDX models are created by implanting fragments of a patient's actual tumor into immunocompromised mice, a process that preserves the tumor’s original architecture, molecular characteristics, and inherent heterogeneity.

Vera emphasizes that traditional, simplified cell lines often fail to capture the complexity of human cancer, which can lead to biased data and an overestimation of drug uptake. To ensure accurate clinical translation, researchers must utilize models that mimic real-world conditions rather than relying on oversimplified systems.

Vera further explores the importance of biodistribution studies, which are fundamental to developing radiopharmaceuticals. By utilizing a larger cohort of PDX models, researchers can account for heterogeneity between different patients. This approach provides a much clearer picture of how a lead compound will behave in a human clinical setting.

As Vera explains, "The more that you involve this patient-derived xenograft, the biggest the cohort or the biggest the number of patient-derived xenograft that you use, even within one disease, is going to give you way better and more information on how your compound will really behave in a real scenario or in the clinic." Ultimately, the interview underscores that the power of PDX models lies in their ability to provide the biologically relevant data necessary for successful clinical translation.

I'm Denis Beckford Vera I am the head of radiopharmacology at Champion Oncology. Patient-derived xenograft model are created by implanting, fragments of real patient's tumor into immunocompromised mice. What is very relevant of this model is that this approach research preserves much of the tumor's original architecture, heterogeneity, and molecular characteristics, which it is very important when you are translating targeted drugs, and in this case, targeted radiopharmaceuticals. Because, as we know, cancer and these diseases are very heterogeneous. And, most of the time, we really use very simplified, tumor models or cell lines that really don't express the real architecture of patient samples. Therefore, using patient-derived xenograft can really mimic what may later, later happen in the clinic, if you are assessing your radiopharmaceutical of your, lead compounds, in patient-derived xenograft. Let's say, rather than mentioning specific examples, I'm going to say in general, when patient-derived xenograft can really, change the development plan or the clinical translation, and again is similar. So if you have, cell lines that have very homogeneous receptor expression, for instance. So you may overestimate your drug uptake or your drug behavior in preclinical models. While if you have patient-derived xenograft, and I like to repeat that because it's very important that it keeps the architecture, the receptor heterogeneity expression, and as well a molecular characteristic of patient samples. Between patients and within a patient, if these samples are taken from the same patient or from different patients, you are really, getting the behavior of the drug that you really need in the, in the clinic. Basically, if you are oversimplifying your model, your animal model, you are basically lying to yourself. So you don't have the opportunity to really see which, the real behavior you sample. More importantly, the patient history. The patient history in those tumors. If you are already targeting a specific disease, you are going to be able to see if a standard of care of other drugs have not been working for that particular tumor or for that particular set of patient populations. So this is gonna be very powerful in translating your radiopharmaceutical, for example. So biodistribution studies are key to developing radiopharmaceuticals. And this is because with radiopharmaceuticals, you have the ability to see. You can treat what you see, and you can see what you treat. So biodistribution studies are probably one of the main components of, developing radiopharmaceuticals. And then we go back to the model. If you are developing, your radiopharmaceutical doing biodistribution, which biodistribution is tied to biology. So if you are oversimplifying your biology, again, you're lying to yourself. You are not really getting the real outcome. You are biasing the outcome. Whether if you use preclinical model with real clinical samples or clinically relevant, animal models, you are really getting the data that you need. The more that you involve this patient-derived xenograft, the biggest the cohort or the biggest the number of patient-derived xenograft that you use, even within one disease, is going to give you way better and more information on how your compound will really behave in a real scenario or in the clinic. You can not only evaluate the heterogeneity of this disease between patients, but also within the patient, if these samples have been taken from the same patient, but from different part of the disease or different part of the patient as well. So it's gonna be very powerful because it is all related to biology.

PharmTech Weekly Roundup–March 13, 2026

Welcome back to the PharmTech Weekly Roundup! I’m Susan Haigney, lead editor of PharmTech.

This week, the industry experienced accelerated digital transformation, regulatory evolution, and a strategic shift toward complex, patient-centric modalities. Across recent developments, we see a clear industry theme: the integration of real-time data and predictive tools—from the FDA’s new adverse event surveillance systems to advanced filling platforms—is being leveraged to reduce manufacturing risks and streamline the path from clinical development to commercial supply. Simultaneously, leadership transitions at FDA could impact long-term regulatory consistency and submission strategies.

FDA Commissioner Marty Makary announced that the director of the Center for Biologics Evaluation and Research, CBER, Dr. Vinay Prasad, will be exiting the agency in April. 

This is Prasad’s second resignation from the agency. He previously left after public criticism but was then reinstated. These frequent leadership changes at FDA may create a volatile regulatory environment that could lead to the reassessment of established submission strategies and clinical evidence expectations.

 was launched by FDA this week. It is a unified platform that consolidates seven fragmented databases into a single dashboard providing manufacturers with real-time access to population-level safety signals. Historical adverse event data will be migrated to the new system, which will contain real-time adverse event reports for all FDA-regulated products by the end of May 2026.

 for an ultra-rare genetic folate deficiency establishes a significant precedent for using real-world and mechanistic evidence to clear the regulatory bar when randomized clinical trials are impractical.

European Commission has approved a 2 mg/kg every-four-weeks dosing regimen for Elfabrio

, offering a significant reduction in cumulative treatment burden for stable adults with Fabry disease.

 have entered a licensing agreement to expand access to cost-effective biosimilars in Latin America by pairing local market expertise with established biologics manufacturing capabilities.

At INTERPHEX this year in New York City, 

Groninger will be debuting a formalized "Lifecycle Solutions" service structure 

and a high-throughput ready-to-use filling platform designed to handle syringes, vials, and cartridges on a single configuration to improve production continuity,. The show is taking place at the Javits Center from April 21 through 23.

, which is happening May 20 through May 22, announced it will host 320 sessions focusing on manufacturing excellence, digital transformation, and the use of predictive tools to reduce technical-transfer risks and accelerate launch readiness.

Check out PharmTech.com for some of our latest technical articles. Contributing editor Cynthia Challener interviewed Tim Heffernan, PhD, vice president and head of the Therapeutics Discovery Division at The University of Texas MD Anderson Cancer Center about 

. The oncology sector is rapidly moving toward precision-driven, complex modalities such as next-generation antibody-drug conjugates, radioligand therapies, and multispecific antibodies to enhance tumor selectivity and overcome the immunosuppressive microenvironment.

A peer-reviewed study by authors from STERIS

 demonstrates that using Raman spectroscopy for identification and a specific detergent additive can effectively remove stubborn Type III magnetite rouge from stainless steel equipment without the need for costly mechanical polishing.

These updates highlight an industry that is becoming more technologically sophisticated while simultaneously managing complex regulatory and global supply chain shifts. Staying aligned with these trends is essential for maintaining compliance and operational efficiency. Visit PharmTech.com to read more about this week’s headlines.

This week, the industry experienced accelerated digital transformation, regulatory evolution, and a strategic shift toward complex, patient-centric modalities. 

Orphan Drug Report 2026 | Created with Canva

On March 13 2026, Evaluate published its 2026 Orphan Drug Report.¹ 

It recounts that rare disease drugs are on track to represent more than 21% of all prescription pharmaceutical sales by 2032, up from 15% in 2022

. That translates to over $400 billion in rare disease drug revenues out of a projected $1.9 trillion total prescription market. This trajectory carries direct implications for pipeline prioritization, capacity planning, and investment decisions.

The report draws on forecasts from Evaluate Pharma to make the case that, despite a turbulent policy environment and intense commercial competition from large-indication products, the rare disease segment has demonstrated consistent resilience.¹ "We continue to see drugs for rare diseases cement their place as major players in the overall drug market,"¹ said Andreas Hadjivasiliou, manager of Content Strategy, Evaluate, in a press release. "Their capacity to navigate a changing industry, policy, and regulatory changes while maintaining momentum is something to be noted by developers in this space and in the mainstream drug markets as well."

What Do Recent Policy Shifts Mean for Rare Disease Development?

The policy landscape has shifted in ways that materially affect the commercial calculus for rare disease programs.¹ Until 2025, only single-indication orphan drugs were exempt from price negotiations under the Inflation Reduction Act. Amendments introduced through the current administration's One Big Beautiful Bill have extended those exemptions to multi-indication drugs. Separately, the Rare Pediatric Disease Priority Review Voucher program was reauthorized in early 2026, restoring an incentive mechanism that promises accelerated review timelines for products targeting rare childhood conditions.

Despite these supportive developments, orphan drugs' share of overall prescription sales is expected to decline modestly during the next seven years.¹ The report attributes this in part to a growing commercial focus on high-volume, large-indication products, most notably obesity treatments, a trend driven by the industry's need to address an estimated $300 billion revenue gap created by approaching patent expirations. This reorientation of Big Pharma resources could affect partnering opportunities, contract manufacturing demand, and competitive dynamics in shared therapeutic platform areas.

On the pipeline and commercial front, the report identifies significant near-term concentration at the top of the market.¹ The eight highest-selling orphan drugs are each forecast to exceed $6 billion in sales by 2032, and the collective sales of today's orphan drug pipeline candidates are projected to surpass $100 billion by that year. Johnson & Johnson's Darzalex, indicated for multiple myeloma, is forecast to be the top-selling orphan drug by 2032, with the subcutaneous formulation of the product credited with effectively doubling the franchise's commercial lifespan. Johnson & Johnson is projected to lead all companies in orphan drug revenues, with nearly $31 billion in forecast sales, placing particular manufacturing and supply chain demands on the organization and its partners.

How Do Recent Law Changes Affect Orphan Drug Exclusivity and Review Times?

Two legislative changes signed into law on February 3, 2026 as part of the Consolidated Appropriations Act have added further regulatory stability to the industry.² Together, they restore and clarify incentive structures that directly affect how developers and manufacturers plan rare disease programs.

The Rare Pediatric Disease Priority Review Voucher program has been reauthorized through September 2029.² Under the program, sponsors who secure approval of a therapy for a rare pediatric disease receive a transferable voucher that reduces FDA review time for a subsequent application from 10 months to approximately 6 months. Given that these vouchers have historically sold for over $100 million, reauthorization restores a meaningful financial lever for organizations weighing investment in rare childhood conditions.

The legislation also resolved a long-running legal ambiguity around orphan drug exclusivity.² A 2021 appellate court ruling had threatened to broaden the exclusivity bar to cover an entire rare disease or condition, rather than the specific approved indication. The new law codifies the FDA's narrower interpretation, confirming that exclusivity applies to the approved use or indication within a rare disease, and does so retroactively.

Evaluate. Evaluate releases 2026 Orphan Drug Report: report reveals that FDA temperament and blockbuster drugs fall short of disrupting the ever-increasing orphan drug market. News release. March 13, 2026. Accessed March 13, 2026.

Wilson Sonsini Goodrich & Rosati. Congress reauthorizes the Rare Pediatric Disease Priority Review Voucher Program and clarifies orphan drug exclusivity. February 9, 2026. Accessed March 13, 2026. 

https://www.wsgr.com/en/insights/congress-reauthorizes-the-rare-pediatric-disease-priority-review-voucher-program-and-clarifies-orphan-drug-exclusivity.html

Rare disease therapies are projected to exceed 21% of global prescription sales by 2032, surpassing $400 billion, underscoring strategic implications for portfolio selection, manufacturing capacity, and capital allocation. Policy updates improve commercial durability: IRA price-negotiation exemptions now extend to multi-indication orphan drugs, and the Rare Pediatric Disease Priority Review Voucher program has been reauthorized. Nonetheless, orphan share may modestly soften as capital shifts toward high-volume franchises (eg, obesity) amid looming patent cliffs. Top-end concentration persists, led by J&J’s Darzalex.

Rare disease drugs are forecast to exceed $400B by 2032, driven by policy shifts and pipeline growth, despite competition from large-indication products.

Why Orphan Drugs Remain Resilient in a Shifting Market

groninger at INTERPHEX NYC 2026 | Created with Canva - Image courtesy of groninger USA

For INTERPHEX NYC, taking place April 21-23, groninger will debut a formalized service structure and put a high-throughput ready-to-use processing platform in front of industry decision-makers.¹ Its presentation will offer a focused look at where filling and closing technology is heading.

What Does a Structured Equipment Lifecycle Program Mean for Manufacturing Teams?

groninger will use INTERPHEX NYC (April 21–23) to introduce its North America Lifecycle Solutions program and demonstrate the UFVN high-throughput ready-to-use (RTU) processing platform. Lifecycle Solutions formalizes end-to-end service support spanning commissioning, optimization, modernization, and retrofit, aligning equipment management with compliance and uptime imperatives. A $14.2 million Charlotte, NC headquarters expansion underpins faster regional response and local manufacturing capacity. UFVN emphasizes 100% in-process control and multi-format capability as prefilled syringes increasingly drive primary packaging strategy and scale-up risk.

groninger will present innovations in equipment lifecycle programs, RTU platform flexibility, and earlier format alignment at INTERPHEX NYC, 2026.

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