How AI can be used in drug development to shorten development timelines.

Pharma Fundamentals: Utilizing Artificial Intelligence in Drug Development

CAI's Jon Thompson details how digital twins cut cleanroom commissioning time by up to 30% and accelerate GMP deviation investigations

Manufacturing Intelligence: How Digital Twins Are Changing Cleanroom Validation

Ask the Expert

This week's headlines saw oncology investment; persistent pressure on supply chain resilience and drug availability; and the importance of operational readiness.

PharmTech Weekly Roundup—June 12, 2026

How evolving regulatory expectations are reshaping pharma development and manufacturing.

Drug Digest April 2026

Navigating FDA Shakeups and Obesity Drug Wars

Behind the Headlines Episode 28

Michael Levy, MSc, MBA is senior vice president, Digital & Innovation, US Pharmacopeia (USP).

Modern digital illustration depicting supply chain challenges in pharmaceutical and medical logistics with interconnected icons | Image Credit: ©KHAN VECTOR CO. -stock.adobe.com

Laboratory scientists, regulatory reviewers, and other professionals responsible for ensuring the quality of medicines are increasingly moving toward digitized workflows. Digital-first tools promise greater efficiency, stronger data integrity, and faster decision-making, yet the reality is its use remains fragmented in many quality environments. Manual steps, such as retyping quality testing instructions, transferring data between systems, or translating standards into formats digital tools can use, continue to slow progress and introduce risk.

As the pharmaceutical industry and quality control labs move toward more connected, data-driven operations, the challenge is not whether to adopt digital technologies, but how to do so while upholding the rigor and confidence that patient safety demands. 

 offer a path forward by embedding established quality expectations for medicines directly into digital environments, helping quality professionals and regulators work more efficiently without compromising control or traceability.

At its core, digitizing quality workflows is about enabling experts to focus on science rather than systems. Making it faster and easier for quality control laboratories and regulators to adopt the tools that help ensure the quality of medicines as workflows evolve is essential. Digital standards are poised to play a critical role by complementing and aligning traditional compendial expectations and modern laboratory technologies to strengthen confidence as more data, methods, and decisions become digital.

 are digitally structured, and machine-readable quality standards. They are designed to carry forward the same science-based rigor as traditional standards in formats that integrate seamlessly into digital workflows.

For experts in quality control labs, this means fewer manual handoffs, less rework, faster method deployment, and clearer traceability across the lifecycle of a method or dataset. For scientists, as well as regulators and reviewers, digitally structured standards can support more consistent standards interpretation and reduced ambiguity on standards implementation to reinforce confidence as regulatory submissions and inspections rely increasingly on digital data.

In quality control labs, the practical impact of digital standards can be significant. When standards integrate into digital workflows, teams spend less time translating text into systems and more time applying scientific judgment. Risk mitigation and traceability are enhanced through electronic capture of steps, version control, and clearer references. Technology transfer can be accelerated through standardized digital workflows and “digital twin” reference materials that help reduce variability across sites and organizations. Automation and advanced analytics become more accessible, freeing resources and supporting continuous improvement. Digital reference standards also avoid some of the inherent limitations of physical materials, such as degradation, logistical or shipping constraints, and environmental impacts associated with solvents or hazardous substances.

From Technology Adoption to Quality Enablement

As quality specialists know, technology alone is not the solution to laboratory digitization. As digital tools evolve rapidly, quality still depends on consistent, trusted standards that can be applied across platforms and organizations. This is where collaboration becomes essential. Enabling adoption of new technologies while upholding quality requires close alignment among method developers, manufacturers, technology providers, and regulators. Digital standards can act as shared, trusted building blocks, helping organizations scale digital workflows with confidence while keeping quality and traceability front and center—from early development of new medicines through commercial manufacturing and delivery.

Progress in developing and implementing digital standards that can make the biggest difference depends on engaging with real-world use cases and learning how new digital technologies perform in operational laboratories. Collaborative development helps ensure that digital standards are practical, fit for purpose, and aligned with regulatory expectations. When industry and regulators contribute early input to these emerging standards, uncertainty may be reduced and adoption may become more feasible, even for organizations with limited resources navigating complex digital transformations.

Quality as a Shared Responsibility in a Digital Era

 (USP), quality is the foundation of a range of solutions being developed to facilitate quality control lab digitization. USP is advancing 

 and related tools to support innovation while preserving the trust placed in compendial standards worldwide.

 By evolving reference standards and documentary methods into validated, digital-first, machine-readable formats, USP is working to integrate quality expectations directly into digitized workflows and to build clear compendial pathways for adopting and scaling emerging technologies—from advanced laboratory informatics to nuclear magnetic resonance analysis software, artificial intelligence, and advanced analytics.

 quality standards framework formally acknowledge that reference standards may be provided as either physical materials or digital data, which is an important step toward reducing uncertainty and strengthening regulatory confidence as digital approaches mature.

 Complementary tools, such as automated software solutions that embed compendial references into analytical workflows, illustrate how digital standards can be applied in practice.

Ultimately, ensuring the quality of medicines in a digital era is a shared responsibility. Digital standards will succeed not because they are new, but because they are trusted, science-based, and developed in collaboration with the community they serve to maximize the value they bring to that community. By working together to align innovation with quality, industry, laboratory scientists, regulators, and other professionals can harness the efficiencies of digital transformation while ensuring quality remains the shared foundation connecting innovation, regulation, and patient care.

Emerging Standards. USP. Accessed June 9, 2026. 

https://www.usp.org/get-involved/emerging-standards-platform

Quality Matters. USP. Published March 24, 2026. 

https://qualitymatters.usp.org/digital-standards-supporting-people-who-protect-quality-medicines-digital-world

Digital Standards. USP. Accessed June 9, 2026. https://www.usp.org/digital-standards

United States Pharmacopeia-National Formulary.

 (USP) is a private, non-profit, scientific organization that develops standards and solutions that help build trust in the supply of quality medicines. Through its rigorous science, USP sets public quality standards that help protect patient safety and improve the health of people around the world. Engage with USP on digital standards by visiting USP’s digital standards 

Articles

Quality organizations are shifting to digital-first laboratory and regulatory workflows, yet manual transcription, data transfers, and format translation still fragment operations and elevate risk. Machine-readable digital quality standards can embed compendial expectations into informatics systems, improving traceability, version control, and consistency of interpretation while accelerating method deployment and technology transfer. Digital reference standards and “digital twin” materials may reduce variability and limitations of physical standards. Broad adoption hinges on collaboration among developers, manufacturers, vendors, and regulators to ensure fit-for-purpose implementation and sustained regulatory confidence.

As quality control labs move toward more connected, data-driven operations, digital technologies must be adopted in a way that upholds patient safety.

Digital Standards Aim to Modernize Quality Assurance without Compromising Confidence

Vice President of Talent Strategies at the Lehigh Valley Economic Development Corporation

Lehigh Valley's deep manufacturing base, community college partnerships, and apprenticeship programs positioned the region to attract Eli Lilly's investment.

Why Eli Lilly Chose Pennsylvania for its $3.5B Facility

Karianne Gelinas, vice president of talent strategies, Lehigh Valley Economic Development Corporation (LVEDC), explains why 

Eli Lilly selected the Lehigh Valley in Pennsylvania for investing $3.5 billion in establishing a new injectable medicine facility.

Gelinas emphasizes that the Lehigh Valley's appeal to Lilly was not accidental. The region hosts 11 colleges and universities, 2 community colleges, and 3 career and technical schools, all operating within an area where manufacturing accounts for 16% of gross regional product.

A pivotal factor in Lilly's decision was the existing partnership between Lehigh Carbon Community College and Wake Tech in North Carolina. Lehigh Carbon had already secured an National Science Foundation (NSF) grant and was planning on beginning the full renovation of its facilities and curriculum when Lilly was evaluating the region. As Gelinas put it, “We weren't doing this reactively. We've been working to strategically attract pharmaceutical and life science companies for years.”

Gelinas also highlights a deeper ecosystem of workforce pathways, including the Industrial Training and Education Consortium, a manufacturing-led apprenticeship nonprofit that has developed five state-approved apprenticeship tracks. Programs in chemistry lab technology, mechatronics, maintenance technology, and quality control are directly applicable to pharmaceutical manufacturing roles. These programs are aiming to supply Lilly's operation with the more than 850 workers the company is expected to bring to the region.

Eli Lilly’s $3.5 billion decision to build a new injectable medicines facility in Pennsylvania’s Lehigh Valley was driven by a mature, prebuilt workforce and training ecosystem. The region combines a strong manufacturing base (16% of gross regional product) with dense higher-education capacity, including multiple universities, community colleges, and technical schools. A preexisting Lehigh Carbon Community College–Wake Tech partnership, supported by an NSF grant and proactive facility/curriculum modernization, signaled readiness. Apprenticeship pathways and aligned technical programs are positioned to staff over 850 anticipated roles.

David O’Connell is director of Scientific and Technical Affairs for PCI Pharma Services.

Abstract luminous DNA molecule. Doctor using tablet and check with analysis chromosome DNA genetic of human on virtual interface. Medicine. Medical science and biotechnology. | Image Credit: ©ipopba -stock.adobe.com

Rare medical conditions can do more than substantially affect wellbeing. They can also make sufferers feel discouraged or even hopeless about their prospects for successful treatments. Because some of these diseases may only impact a handful of people worldwide, rare diseases bring an inherent lack of scale—a dearth of established research and infrastructure that make journeys to effective treatments that much longer and more arduous.

Their sheer diversity only exacerbates this difficult dynamic. True to their categorization, rare diseases are as inconsistent as they are infrequent. Typically genetic and often severe, such conditions span a wide spectrum of therapeutic areas, from oncology and neurology to immunology and metabolic disease. The result is a “broad but not deep” scenario that, unfortunately, can leave treatment resources as scarce as the diseases themselves.

Worse yet, “rare” is both a formal designation and a subjective, relative term. While some rare conditions may indeed be one-in-a-million misfortunes, others are far more prevalent. Officially, the threshold in the world’s two most developed markets—the United States and Europe—define rare conditions as those presenting in less than 1 in 2000 people. (Europe has an official 2000:1 ratio

, while the US sets a nationwide limit of 200,000

, which creates a comparable ratio given its population of over 340 million.) Considering this, even rare diseases can cause widespread suffering and unmet treatment needs affecting millions of patients worldwide.

Serving this unconventional global community demands a suitably unconventional pharmaceutical model: one that prioritizes precision, integration, and geographic reach rather than scale. Promisingly, these attributes increasingly align with those displayed by modern contract development and manufacturing organizations (CDMOs)—partners that, at their most comprehensive, are equipped to take the highly specialized molecules used to treat rare diseases from early phase clinical trials through commercial reality.

Precision Molecules for Precision Populations

From a development and processing perspective, treatments for rare diseases demand a rarified air of pharmacological prowess. Unlike therapies for more common conditions, which often rely upon well-understood molecules with broader applicability, many rare disease programs involve highly targeted agents tailored to distinct genetic or molecular signatures. Unsurprisingly, a substantial portion of today’s rare disease and targeted therapies are highly potent compounds, reflecting a broader industry trend in which highly potent APIs represent roughly 30% of drug development pipelines,

and are particularly prevalent in precision medicines.

Aligning with this paradigm shift, it isn’t unusual for rare disease investigational pipelines to be dominated by biologics, gene-based modalities, or other specialized molecular classes. Here, biology matters: roughly 80% of rare conditions have a genetic component,

 making them amenable to molecularly directed approaches such as antisense oligonucleotides, enzyme replacements, or targeted inhibitors that modulate specific pathways or proteins. Such modalities often require sophisticated formulation, containment, and dose control far beyond standard therapies. Encouragingly, these elevated requirements are reflected in the pharma industry’s shifting priorities: industry analyses suggest that approximately 35% of all drugs and biologics currently in global R&D pipelines are intended to treat rare diseases.

For developers and their manufacturing partners, molecular intricacy translates into complexity: APIs that are potentially highly potent, bespoke biologic processes, and the need to balance stability, safety, and efficacy in exceedingly small patient populations. It’s a landscape where scientific nuance is inseparable from operational execution.

Structural Considerations for Rare Disease Programs

Rare disease drug development not only operates at smaller scales, but also under differing sets of physical, regulatory, and human constraints. Hard facts speak to the challenges such fragmentation poses: though there are more than 7000 recognized rare diseases worldwide

—collectively affecting approximately 300 million people—fewer than 5% of these conditions have an FDA-approved treatment.

In conventional pharma, large patient populations allow for trial delays, formulation iterations, and wide-scale manufacturing tolerances. With rare diseases, no such leeway exists. A single trial site may represent a significant proportion of the available patient pool, which means missing a shipment can meaningfully delay or derail an entire study. In fact, clinical trial enrollment itself presents a structural challenge, with rare condition studies seeing significantly fewer participants than common disease trials. The rarer the condition, the scarcer investigational evidence can become; many rare diseases have seen fewer than 10 trials, placing a premium on every opportunity to accrue sound research.

Geography can further complicate matters. Rare disease patients are frequently scattered across multiple regions, compelling sponsors to navigate multi-jurisdictional regulatory frameworks and logistics for very small treatment volumes. And because these therapies often involve highly potent, targeted molecules or genetically informed modalities demanding precise formulation and delivery, processing and logistical fragmentation becomes another potential pitfall. Successful rare disease treatment development requires the synchronized orchestration of science and supply chain that forgo mere scale in favor of agility, adaptability, and global reach.

Considering this, the central challenge posed by rare disease treatment is not how much medicine can be manufactured, but how exactingly it can be produced, released, and delivered. Volumes are often measured in hundreds or even dozens of units, yet those units must reach patients dispersed across multiple countries under tightly controlled regulatory and quality frameworks. A single clinical or commercial batch may represent a meaningful share of the global supply, leaving no room for deviation in formulation, sterility, or containment.

This reality places unusual demands on manufacturing infrastructure. CDMOs supporting rare diseases must be capable of low-volume, high-mix production, where frequent changeovers, short campaigns, and evolving formulations are the norm rather than the exception. For many rare disease therapies—such as highly potent small molecules and sterile injectables—this painstaking process must unfold within advanced containment systems designed to protect both product integrity and operator safety, often with exposure limits of mere nanograms.

In turn, those exceptionally limited batches must be packaged, labeled, released, and distributed into a multi-region clinical and commercial supply network, supporting trials and patients across North America, Europe, and beyond. Such scenarios are where integration becomes decisive. When development, oral solid dose and sterile manufacturing, quality, and global distribution sit within a single CDMO framework, rare disease sponsors gain something more valuable than scale: continuity of control from first patient to final dose.

Consider a theoretical rare disease therapy entering first-in-human trials with fewer than 20 patients worldwide. The biology is specific, the therapeutic window narrow, and the doses measured in micrograms rather than milligrams. For both the patients and the sponsor, this creates a set of demands that are fundamentally different from conventional pharma production practices. The program must accommodate the following:

A globally dispersed patient population, requiring clinical supply into multiple regions from the very first study

Extremely small cohort sizes, where each dose and batch carry outsized importance

The likelihood of a highly potent, targeted active ingredient demanding advanced containment and precise dose control

A compressed development pathway where early pharmacokinetic and safety data must be generated expediently to avoid impacting characteristically scarce patient enrollment

A need for low-dose first-in-human material, often enabled through micro-dosing platforms such as Xcelodose.

Meeting these demands requires a manufacturing and supply infrastructure built for meticulousness rather than volume. Early clinical material is produced using controlled micro-dosing systems that deliver uniformity at the microgram level, protecting patients while generating reliable data. As the program progresses, the same formulation and containment strategies are carried forward into larger batches, preserving continuity and avoiding disruptive redevelopment.

At the same time, clinical trial supply becomes a global exercise: packaging, multi-language labeling, regulatory release, and distribution into regional depots all must be coordinated for low-volume, high-value shipments. Digital supply platforms track site-level demand and inventory in real time, ensuring that no patient misses a dose due to forecasting error or logistical delay. By the time the therapy reaches late-stage development, the operational framework supporting it is already aligned for commercial reality.

Rare disease medicine now sits at the intersection of scientific possibility and industrial responsibility. The tools to design targeted therapies exist, and the pipelines are increasingly rich with orphan and precision assets. What determines whether these advances translate into real outcomes for patients is no longer discovery alone, but the infrastructure that surrounds it.

Rare disease pipelines eschew pharmaceutical models built around volume and scale, in favor of those prioritizing precision, continuity, and global coordination. Success depends on moving seamlessly from laboratory insight to manufacturable product, from early clinical material to long-term commercial supply, and from fragmented efforts to integrated operational systems.

Of course, these prerequisites are largely invisible to patients. For them, all that matters is whether therapies are viable, effective and available. Behind that sits a complex machinery of formulation science, containment engineering, regulatory alignment, and global distribution—all designed to serve populations too small to benefit from conventional supply-and-demand economics.

In that sense, rarity is no longer just a biological category. It is a measure of how willing the industry is to build systems for those who cannot be served by scale. Not because it is easy, but because the science, the infrastructure, and the ethical imperative have converged. This is a rare moment indeed—one that must be seized by pharma companies and the integrated CDMOs they entrust to help develop, produce, and distribute life-saving rare disease treatments.

Health Europe. Rare diseases. European Commission. Accessed June 15, 2026. 

https://health.ec.europa.eu/rare-diseases-and-european-reference-networks/rare-diseases_en

Rare diseases at FDA. FDA. Accessed June 15, 2026. 

https://www.fda.gov/patients/rare-diseases-fda

Small molecule API market size, share & industry analysis, by type. Fortune Business Insights. Updated May 25, 2026, 

https://www.fortunebusinessinsights.com/small-molecule-api-market-107457

Hyun JB, Nam Y, Rim YA, et al.. Advances in rare disease therapy for rare disease treatment. 

https://pmc.ncbi.nlm.nih.gov/articles/PMC12658154/

Getz K, The Hard Truth About Rare Disease and Gene Therapy Drug Development,” 

https://www.appliedclinicaltrialsonline.com/view/the-hard-truth-about-rare-disease-and-gene-therapy-drug-development

First-ever rare diseases resolution underscores equity and inclusion.WHO. May 24, 2025, 

https://www.who.int/news/item/24-05-2025-seventy-eighth-world-health-assembly---daily-update--24-may-2025

https://globalgenes.org/rare-disease-facts/

David O’Connell, BSc (Hons) is Director of Scientific and Technical Affairs for PCI Pharma Services

a leading CDMO providing integrated end-to-end drug development, manufacturing and packaging capabilities that increase products’ speed to market and opportunities for commercial success. In his current role, O’Connell aids clients with formulation development, technical transfer and scale-up of solid oral, oral liquid and semi-solid products for clinical trials and/or commercialization. 

Rare diseases, though individually uncommon, collectively affect hundreds of millions and remain largely untreated, with fewer than 5% having FDA-approved therapies. Development increasingly relies on highly potent small molecules, biologics, and gene-based modalities aligned to genetic etiologies and narrow therapeutic windows. The limiting factor is not volume but precision—microgram dosing, stringent containment, and defect-intolerant quality systems—coupled with globally dispersed patients and multi-jurisdictional regulation. Integrated CDMO platforms that unify development, manufacturing, packaging, release, and distribution can de-risk trials and enable seamless commercialization.

Treatment pipelines for rare diseases have never been more promising. Optimizing their impact will require integrated, precision-first CDMO support systems.

Optimizing Impact for Rare Diseases

PharmTech Weekly Roundup--June 12, 2026

This week's headlines saw a wave of high-stakes oncology investment as companies race to secure next-generation therapeutic platforms; persistent pressure on supply chain resilience and drug availability; and the growing importance of operational readiness—whether that means data infrastructure for AI, carbon accountability, or access systems for complex modalities like radiopharmaceuticals.

 for $10.6 billion, gaining two late-stage kinase inhibitors targeting ROS1 and ALK alterations in non-small cell lung cancer—both carrying FDA Breakthrough Therapy designations with potential approval decisions later this year.

 have committed a combined $4 billion to protein degrader platforms—J&J acquiring Firefly Bio for its tumor-targeted degrader conjugate technology, and Roche licensing Nurix's BTK degrader bexobrutideg—signaling that targeted protein degradation is becoming a primary pipeline priority in oncology.

European Medicines Agency published its 2025 annual report

, which detailed the agency’s approval of 104 human medicines in 2025, including 38 new active substances and several first-in-class therapies, while also advancing a draft policy that could reduce the clinical data burden for biosimilar development.

US Pharmacopeia’s 2025 annual drug shortages report

 found that while the total number of shortages declined by 23% in 2025, drug discontinuations rose 60% year-over-year—with low-priced generics and sterile injectables at highest risk, pointing to structural fragility in the supply base.

Nicholas Richardson, vice president of clinical development at Precision for Medicine,

 explained how radiopharmaceuticals require an integrated system of isotope production, just-in-time distribution, nuclear medicine licensing, and trained personnel that makes patient access inherently complex—and developers must build site strategy and supply chain infrastructure into development planning from the outset.

 argues that companies must move beyond reactive tariff responses and build documented, quality-led supply chain resilience strategies — including dual sourcing and qualified backup suppliers — that can withstand FDA scrutiny and protect patient access.

 explain how pharmaceutical organizations should evaluate three clinical data architectures — data warehouses, data lakes, and data lakehouses — based on their AI readiness, governance requirements, and technical capacity, with the lakehouse model offering the most comprehensive capabilities for large research-intensive institutions.

David Vázquez, climate principal consultant, Nexius Projects,

 spoke about how pharmaceutical suppliers face growing pressure to calculate and report product-level carbon footprints as major customers like AstraZeneca, the UK NHS, and regulators in France begin mandating emissions data—with experts noting that carbon accounting also surfaces manufacturing inefficiencies that can reduce costs.

Thank you for watching. Be sure to read all the news and feature content on PharmTech.com.

Videos

Senior Director
Education, Outreach, and Digital Experience
Open Biopharma Research and Training Institute

, co-hosts Chris Cole and Richard Jaenisch explore the emerging role of digital twins in cleanroom design and qualification in pharmaceutical manufacturing with guest Jonathon Thompson, global director of the digital center of excellence at CAI.

To view the full fascinating, practical, engaging conversation, click here!

Digital twins of cleanrooms are offering measurable reductions in commissioning time, rework, and pressure excursion investigation, but only when paired with rigorous real-time data verification, explains Thompson. He adds that the technology's core value lies not in replacing IQ/OQ/PQ, but in catching design flaws before construction locks them in.

The traditional cleanroom qualification workflow exposes problems late, Thompson notes, often after equipment is already installed and early-phase processes are running. Digital twins, fed with 3D building information modeling (BIM) models, HVAC specifications, personnel flow data, and physics-based fluid dynamics simulations, allow manufacturers to stress-test facility designs upstream. Thompson points to tangible returns: 10–30% reductions in commissioning time and resource hours, 20–50% reductions in rework, and 30–70% faster root cause identification during pressure excursions.

Regulatory acceptance, Thompson argues, depends on how the twin is used. "If you're using this digital twin as your single source of truth without verifying that data in real time with what's actually happening out in the field," he warns, "they're going to start to question it." Continuous process verification—cross-referencing the model against live environmental monitoring data, particle counts, and pressure differentials—is what earns FDA and EMA confidence in the approach.

For CDMO and modular facilities, where equipment configurations and product mixes change frequently, the stakes around model maintenance are especially high. Thompson observes that "we've got to change the mindset" to treat the BIM model as a living asset subject to change management, not a static deliverable from the capital project phase. As generative AI and light detection and ranging-based reality capture mature, automated model updating may eventually reduce that burden, making the digital twin a true operational constant rather than a commissioning tool.

CAI's Jon Thompson details how digital twins cut cleanroom commissioning time by up to 30% and accelerate GMP deviation investigations.

Vacuoles, E1 enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) syndrome

GSK has secured Orphan Drug Designations from both the FDA and the EMA for momelotinib in the treatment of Vacuoles, E1 enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) syndrome, a rare and severe hemato-inflammatory condition for which no approved therapies currently exist.

 The dual designation marks a meaningful step in expanding the therapeutic profile of a drug that is already approved for myelofibrosis across several major markets.

VEXAS syndrome is a clonal myeloid disorder that sits at the intersection of rheumatology and hematology.

 Clinically, it presents with a broad spectrum of inflammatory manifestations, including prolonged fever, relapsing chondritis, vasculitis, and lung involvement, alongside hematologic complications such as macrocytic anemia, thrombocytopenia, and progressive bone marrow failure that can evolve into hematologic malignancy. Caused by a somatic mutation in the UBA1 gene located on the X chromosome, the condition predominantly affects men over 50. Its prognosis is poor: five-year mortality sits between 30 and 40%, yet the disease remains without any approved treatment options. The designations were supported by retrospective case data suggesting that Janus kinase inhibitors may offer therapeutic benefit in VEXAS, along with a case report indicating clinical improvement with momelotinib specifically, including reductions in disease-related inflammation and hematological manifestations.

What Does This Mean for Momelotinib's Development Program?

Momelotinib's mechanism sets it apart from other Janus kinase inhibitors.

 The compound inhibits JAK1 and JAK2, pathways implicated in constitutional symptoms and splenomegaly, while also targeting activin A receptor type I, which reduces circulating hepcidin levels and contributes to anemia benefit. That triple-pathway inhibition is central to why the molecule is attracting attention beyond its current indication.

The drug is already approved in the US for intermediate or high-risk myelofibrosis in adults with anemia and holds approvals in the EU, UK, and Japan for myelofibrosis with disease-related splenomegaly, symptoms, or moderate to severe anemia.

 The VEXAS program represents a deliberate expansion into adjacent hematological territory.

GSK has initiated planning for the ATLAS phase II/III trial, which will evaluate momelotinib's efficacy and safety in VEXAS syndrome and serve as the foundation for planned global regulatory submissions.

 The study design is scheduled to be presented at the 2026 European Hematology Association Congress in June.

Orphan drug status in both the US and EU confers regulatory and commercial incentives, including extended market exclusivity and reduced filing fees, that influence how companies prioritize and resource development programs.

 For a molecule already navigating post-approval lifecycle management in myelofibrosis, these designations reinforce the case for continued manufacturing investment and supply chain planning.

The VEXAS indication also introduces formulation and development considerations.

 As a rare disease with a predominantly older male patient population and complex hematological presentation, any eventual commercial program will require close coordination between clinical, regulatory, and manufacturing teams well ahead of any potential approval. The ATLAS trial design, when presented at the European Hematology Association Congress, will be worth monitoring for signals that could shape those downstream requirements.

How Does This Fit into GSK's Broader Hematology & Oncology Push?

The VEXAS designations arrive as GSK pursues an aggressive expansion across hematology and oncology.

The company recently announced a $10.6 billion agreement to acquire Nuvalent

, a clinical-stage biopharmaceutical company with two late-stage non-small cell lung cancer assets, zidesamtinib and neladalkib, both currently under FDA review with potential 2026 approval decisions. The deal also includes a phase I HER2 inhibitor and a preclinical portfolio built around precision medicine. Together with the momelotinib VEXAS program, the moves signal a deliberate strategy of targeting conditions where existing therapies carry meaningful efficacy or tolerability limitations.

GSK plc. GSK’s momelotinib granted orphan drug designations in the US and EU for VEXAS syndrome. Press Release. June 11, 2026. 

https://www.gsk.com/en-gb/media/press-releases/gsk-s-momelotinib-granted-orphan-drug-designations-in-the-us-and-eu-for-vexas-syndrome

GSK plc. GSK enters agreement to acquire Nuvalent, Inc. Press Release. June 8, 2026. 

https://www.gsk.com/en-gb/media/press-releases/gsk-enters-agreement-to-acquire-nuvalent-inc/

GSK obtained FDA and EMA Orphan Drug Designations for momelotinib in VEXAS syndrome, a UBA1 somatic mutation–driven hemato-inflammatory disorder with high mortality and no approved treatments. Evidence includes retrospective experience suggesting JAK inhibition benefit and a momelotinib case report showing improvement in inflammatory and cytopenic features. Momelotinib’s JAK1/2 and ACVR1 inhibition may address symptoms, splenomegaly, and anemia via hepcidin reduction. Planning has begun for the ATLAS phase II/III trial, with design details expected at EHA 2026. Orphan incentives support lifecycle investment and manufacturing planning in rare disease.

GSK's momelotinib receives US and EU orphan drug designations for VEXAS syndrome, a rare myeloid disorder with 30–40% five-year mortality and no approved treatments.

GSK's Momelotinib Pursues Rare VEXAS Syndrome Indication

 connected with Tom Sellig, CEO, Adare Pharma Solutions, to discuss manufacturing investment trends, 3D-printed tablets, FDA dynamics, and the workforce strategies shaping the company's growth.

Tom Sellig on CDMO Innovation and US Manufacturing Growth

PharmTech: What Technologies Are Most Central to Adare's Innovation Strategy?

 Patient-centric dosage forms are core to our model, and 3D printing of tablets is one of the most exciting areas driving our growth. The technology allows us to differentiate product formulations and produce a wide range of dosage forms and release profiles in ways that more conventional manufacturing approaches cannot easily match. We're also paying close attention to the ongoing conversation around solubility and bioavailability enhancement. While it's an important capability to have, we think many companies are still working within the boundaries of existing technologies. We're developing what we believe are novel solutions that could leapfrog some of what's currently available in the market.

How Are Shifts at the FDA Affecting Your Development Timelines?

The FDA is going through significant changes right now, and that's creating a genuinely different dynamic between CDMOs [contract development and manufacturing organizations] and pharma companies. The conversation is increasingly about how to accelerate timelines while still working within the regulatory framework. We're actively supporting products that have received priority review vouchers, and that creates real pressure to move faster. It's a challenge, but it's also an opportunity for us to demonstrate the value of a CDMO that has the technical depth to execute under that kind of pressure.

How Is Adare Approaching Talent Acquisition and Retention?

Talent is a critical priority for us. We have an internal acquisition team, but we're also building something we call the Adare Academy, a structured, skills-based development and training program for all employees. It covers both soft skills and hard skills, including science and technology, with an explicit focus on career development pathways. The goal is not just to attract people, but to retain them. Lower turnover translates directly into continuity for our teams, for our client relationships, and ultimately for service quality.

How Do You View the Wave of Reshoring Commitments from Large Pharma, and What Does It Mean for CDMOs?

The scale of investment being discussed is significant. That said, these are long-horizon projects. Whether all of them come to fruition, and on what timeline, remains to be seen. Our response is to focus on what we can act on today: building out selective capacity and capabilities that position us to support the industry's growth as those investments materialize. We don't need to wait for the full wave, we need to be ready when it arrives.

Adare Pharma Solutions is prioritizing patient-centric dosage forms, with 3D-printed tablets enabling differentiated formulations and tailored release profiles beyond conventional manufacturing. The company is also pursuing next-generation solubility and bioavailability approaches intended to surpass current market options. FDA operational shifts are intensifying sponsor–CDMO collaboration around accelerated development, particularly for programs leveraging priority review vouchers. Growth plans emphasize workforce stability via a structured internal training and career-pathway platform. Reshoring commitments are viewed as meaningful but long-cycle, driving selective near-term capacity buildout.

Tom Sellig, Adare, discusses 3D-printed tablets, US reshoring investment, FDA timeline pressures, and workforce development strategies driving CDMO growth.

Q&A: Tom Sellig on Why Complex Formulations Are Driving CDMO Growth

Jordan Xu is an engineering expert specializing in automated pharmaceutical manufacturing systems. He is currently with DROFEN MACHINERY EQUIPMENT CO., LTD, a leading system supplier of turnkey automated assembly lines for insulin pens and aseptic filling systems for PFS. With extensive experience in resolving fill-finish bottlenecks, DROFEN partners with global CDMOs to deliver GMP-compliant, high-speed production solutions that accelerate time-to-market.

Transitioning to fully automated assembly lines

The global shift toward self-administration of biologics has driven the exponential demand for pen injectors.

 For pharmaceutical manufacturers and CDMOs, scaling up production to meet this demand requires robust automated assembly lines. While individual machines may boast high theoretical throughput, the overall equipment effectiveness (OEE) often suffers due to micro-stoppages, component variations, and integration complexities between pre-assembly and final assembly stages.

Addressing these bottlenecks requires a holistic systems-engineering approach, focusing on the seamless integration of pre-filled syringe (PFS) handling, dosing mechanism assembly, and rigorous quality control at high speeds.

Challenge 1: Component Variability and High-Speed Feeding

One of the primary causes of micro-stoppages in high-speed assembly lines, operating at 100-160 parts per million (PPM), is the dimensional variability of molded plastic components.

 Even minor deviations within acceptable tolerances can lead to jamming in vibratory bowl feeders or misalignment during robotic pick-and-place operations.

 To mitigate this, advanced assembly lines must employ adaptive feeding systems. Utilizing vision-guided robotics for component orientation rather than relying solely on mechanical sorting significantly reduces jam rates. Furthermore, implementing continuous-motion assembly architectures, in which components are assembled on a moving turret rather than intermittent indexing systems, provides smoother handling of fragile parts and accommodates slight dimensional variations without halting production.

Challenge 2: Precision Alignment in Final Assembly

The final assembly of a pen injector, in which the drug cartridge or pre-filled syringe is integrated with the dosing mechanism, is the most critical phase. The alignment must be perfect to ensure the dose accuracy and the mechanical integrity of the device. Misalignment not only damages the glass cartridge, leading to costly product loss, but also poses severe safety risks to the end-user.

 Achieving precision at 160 PPM requires moving beyond standard pneumatic actuators. Servo-driven pressing stations equipped with force-displacement monitoring provide the necessary control.

 By continuously monitoring the force profile during the pressing operation, the system can detect anomalies, such as a slightly oversized cartridge or a misaligned thread, in milliseconds. If the force profile deviates from the validated parameters, the system can abort the operation before glass breakage occurs, rejecting the specific unit without stopping the entire line.

Challenge 3: Seamless Integration of Pre-Assembly and Final Assembly

Many manufacturers procure pre-assembly equipment and final assembly equipment from different vendors. This fragmented approach often leads to integration bottlenecks in which the transfer of sub-assemblies between machines creates buffer accumulation issues and increases the risk of contamination or damage.

 A turnkey approach, in which a single system supplier provides both the pre-assembly and final assembly lines, eliminates these integration hurdles. This allows for a unified control architecture (e.g., a single PLC/HMI platform) and seamless physical transfer systems. By designing the line as a cohesive unit, manufacturers can implement intelligent buffering systems that dynamically adjust the speed of upstream processes based on downstream demand, maintaining a continuous flow and maximizing overall equipment effectiveness.

Challenge 4: Ensuring Compliance and Accelerating Validation

In the highly regulated pharmaceutical industry, high-speed production is meaningless without stringent quality assurance and comprehensive validation (installation qualification/operational qualification/performance qualification).

Implementing 100% in-line inspection at 160 PPM generates massive amounts of data, which can overwhelm standard control systems.

 Modern assembly lines must integrate edge computing to process vision inspection data locally, ensuring real-time pass/fail decisions without latency.

Furthermore, adopting a quality by design approach during the engineering phase ensures that critical process parameters are continuously monitored and controlled.

 Partnering with equipment suppliers who offer comprehensive validation documentation and regulatory support significantly accelerates the factory acceptance testing/site acceptance testing processes and overall time-to-market.

Scaling up pen injector production to meet global demand requires overcoming significant engineering bottlenecks in component handling, precision assembly, and system integration. By adopting advanced servo-control, vision-guided feeding, and a unified turnkey approach to pre-assembly and final assembly, manufacturers can achieve stable, high-speed production. Partnering with an experienced system supplier who understands both the mechanical intricacies and the stringent regulatory requirements is essential for ensuring product quality and accelerating time-to-market in this competitive landscape.

, a turnkey system supplier of automated assembly lines for injection pens and pre-filled syringes for global pharmaceutical manufacturers and CDMOs."

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Applying lean methodologies to improve the OEE of a manufacturing line: a case study in a pharmaceutical company

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Efficient force-displacement monitoring and process control for medical device assembly

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GAMP 5: A Risk-Based Approach to Compliant GxP Computerized Systems

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https://doi.org/10.1007/s10668-023-03225-8

Surging global adoption of self-administered biologics is forcing rapid scale-up of pen-injector manufacturing, where realized throughput is constrained by OEE losses from micro-stoppages, part variability, and handoffs between pre- and final-assembly modules. Key failure modes include molded-component dimensional drift that disrupts high-speed feeding, tight alignment tolerances during PFS/cartridge integration, and vendor-fragmented line architectures that create buffering and contamination risks. Mitigations center on vision-guided adaptive feeding, continuous-motion platforms, servo pressing with force–displacement analytics, turnkey integrated controls, and edge-computing-enabled inline inspection aligned with QbD and GxP validation.

GLP-1 / insulin demand is straining CDMO fill-finish capacity. This piece examines bottlenecks in automated pen injector assembly and strategies to optimize operations.