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

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Closing the Gap in Modern Process Control and Automation for QC/QA

Drug Digest December 2025

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A “Plan, Prevent, Prove” Approach

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Pharmaceutical compounding, particularly in 503A and 503B environments, involves complex formulation and regulatory challenges far beyond academic theory. This summary of 

 on this topic—written by industry professionals Paul Pluta RPh, PhD, Alan Mancini RPh, Nishant Thakar, RPh, PharmD, and Varanya Chaiyaperm, RPh, PharmD—distills the essential technical and operational takeaways from a comprehensive, six-stage model of pharmaceutical compounding. Ideal for professionals in drug development, manufacturing, QA/QC, and regulatory compliance, this summary emphasizes real-world application, risk mitigation, and alignment with quality frameworks like Quality by Design, Good Manufacturing Practices (GMP), and Quality Risk Management (QRM).

Stage 1: design the compounding blueprint

Establish a complete plan including ingredients, packaging, calculations, labeling, and patient-specific factors.

Integrate regulatory standards (US Pharmacopeia, FDA bulk lists, GMP) and proactively identify risks using QRM tools.

Ensure aseptic setup, verified materials, and technician preparedness—especially critical for sterile and high-risk compounds.

Require dedicated or disposable equipment for hazardous or cross-contaminating drugs (e.g., antineoplastics, allergens).

Stage 3: dosage form completion & quality evaluation

Conduct rigorous physical and analytical testing; 503B batches must meet GMP-level standards.

Finalize documentation with full traceability and ensure preparations are quarantined until verified.

Stage 4: packaging & patient-ready delivery

Assemble patient/client information equivalent to commercial products. Ensure all packaging, labeling, and delivery logistics meet regulatory and clinical standards.

Implement safeguards for temperature control and light protection during distribution.

Stage 5: independent verification before release

Engage an independent observer to verify formulation integrity, test results, documentation, and labeling accuracy.

Use checklists to prevent critical errors and maintain consistent approval protocols.

Stage 6: dispensing, distribution & final safety checks

Dispense only after final pharmacist approval, ensuring two-point patient ID verification.

Monitor for common hazards like name mix-ups, transcription errors, and shipping delays.

Management takeaways: quality starts at the top

Leadership must prioritize hiring, SOP adherence, continuous training, and fostering a quality-driven culture.

Management bears ultimate responsibility for SOP execution, staff performance, and patient safety outcomes.

The six-stage compounding model offers a structured, risk-aware approach to compounded drug development. For pharmaceutical manufacturing professionals, it ensures regulatory alignment, enhances process reproducibility, and reinforces a culture of quality and accountability across the compounding lifecycle.



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A structured, risk-based framework for pharmaceutical compounding that enhances quality, regulatory compliance, and process consistency.

The 6-Stage Compounding Framework Every Pharmaceutical Manufacturing Professional Should Know

Kyiv, Ukraine - December 22, 2020: Backlit single shot image of GlaxoSmithKline logo on tv screen with a hand holding an Covid-19 vaccine concept | Image Credit: © Олександр Луценко - stock.adobe.com

FDA has approved a prefilled syringe version of GSK’s Shingrix, a recombinant zoster vaccine used to prevent shingles, eliminating the need for reconstitution of separate vials prior to administration (1). The newly approved format aims to ease preparation for healthcare professionals, enhancing efficiency and reducing the risk for handling errors during vaccination.

The previous formulation of the shingles vaccine required clinicians to combine a lyophilized antigen with a liquid adjuvant prior to administration (1). FDA’s approval of the new single-syringe version follows data confirming its technical comparability with the original two-vial presentation.

·FDA approval of the prefilled syringe version of the recombinant zoster vaccine supports manufacturing shifts toward ready-to-use, single-dose formats.

·Technical comparability data enabled regulatory clearance without reformulation, highlighting the value of platform consistency in vaccine development.

·Prefilled syringe design may enhance uptake by reducing administration errors and simplifying logistics across healthcare and immunization programs.

Brigid Groves, vice president of professional affairs at the American Pharmacists Association, emphasized the practical benefits of this update (1): “The prefilled syringe presentation of GSK’s shingles vaccine is good news, providing a convenient method of administration. The FDA approval is a positive step toward driving prevention of this painful disease, and as a practicing pharmacist I welcome the availability of this new presentation.”

Expanded access and continued eligibility guidelines

As with the original version, the prefilled syringe formulation is licensed in the United States for immunization of adults aged 50 and older, as well as individuals aged 18 and older who are or will be immunocompromised due to disease or medical treatment (1). Current guidance from the Centers for Disease Control and Prevention recommends a two-dose series of the vaccine for these groups to help prevent shingles and its complications.

Tony Wood, chief scientific officer at GSK, commented on the public health rationale for the updated presentation, stating “at GSK, we are committed to advancing scientific innovation and delivering practical solutions that address the needs of the healthcare community. This new presentation of Shingrix was developed to streamline the vaccination process, supporting healthcare professionals to provide protection against shingles, a disease that one in three US adults will develop in their lifetime” (1).

In parallel to the US approval, the prefilled syringe presentation is under review by the European Medicines Agency, with the application accepted for evaluation in January 2025 (1). Regulatory submissions are also being considered for additional international markets.

These developments reflect a broader industry focus on increasing adult immunization rates through simplified vaccine formats.

Shingles, also known as herpes zoster, is a painful skin rash caused by reactivation of the varicella-zoster virus (VZV)—the same virus responsible for chickenpox (2). While not all individuals with latent VZV will develop shingles, nearly 99% of US adults over 50 harbor the virus, and about one million cases are diagnosed annually in the US (3).

With age, immune function diminishes, increasing the likelihood that the virus will reactivate (4). This makes older adults more susceptible to shingles and its associated complications, including postherpetic neuralgia, a condition characterized by long-lasting nerve pain.

The recombinant zoster vaccine is a non-live, subunit formulation that combines glycoprotein E with the proprietary AS01B adjuvant system (1). It is designed to trigger a strong immune response even in older adults, whose immune systems may respond less robustly to vaccination. The vaccine is not intended to prevent chickenpox or primary varicella infection.

The newly approved Shingrix format comes just days after GSK announced that FDA had accepted GSK’s application to extend the indication for the company’s respiratory syncytial virus (RSV) vaccine, adjuvanted (Arexvy) to adults aged 18–49 who are at increased risk (5). The vaccine is approved in the US to prevent lower respiratory tract disease (LRTD) caused by RSV in adults older than 60 years. It is also approved to treat people aged 50–59 who have increased risk for LRTD caused by RSV.

US FDA Approves GSK’s Shingrix in a Prefilled Syringe Presentation. Press Release

Harpaz, R.; Ortega-Sanchez, I.; Seward, J.; et al. 

Prevention of Herpes Zoster: Recommendations of the Advisory Committee on Immunization Practices (ACIP)

Varicella Zoster Virus Infection: Clinical Features, Molecular Pathogenesis of Disease, and Latency

GSK Seeks Expanded Use Approval from FDA for RSV Vaccine Arexvy

FDA Approves Shingrix Prefilled Syringe to Simplify Vaccine Administration

Tore Bergsteiner summarizes the promise of AI

AI Boosts Early Disease Detection and Diagnostic Accuracy

 back in March 2025, Tore Bergsteiner, managing partner at MAIN5, a European consulting firm specializing in digitally enabled change for life sciences R&D organizations, discusses how AI is increasingly enhancing diagnostics and pathology by enabling early disease detection through advanced image analysis, improving treatment outcomes and patient safety. He also notes how AI helps address global shortages of pathologists by boosting diagnostic efficiency and accuracy. Both points still hold true 4 months later.

Check out our full interview with Tore 

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Tobin bridge, Zakim bridge and Boston skyline panorama at sunset | Image Credit: © mandritoiu - stock.adobe.com

The 15th annual Partnership Opportunities in Drug Delivery (PODD) conference, set for October 27–28, 2025, at the Westin Copley Place in Boston, will spotlight the latest developments in drug delivery technology with a program of expert panels, presentations, and industry-driven sessions (1).

Organized by the Conference Forum, the event brings together pharmaceutical developers, biotech innovators, and drug delivery specialists to examine advances in the formulation, design, and commercial deployment of therapeutics across a range of modalities (1).

Focus on strategic partnering in drug delivery

·Drug delivery partnerships are evolving, with top pharma leaders outlining strategic evaluation frameworks for external technologies and collaboration models.

·Pharma innovation leaders are refining internal scouting, sharing insights into sourcing enabling technologies that accelerate drug development pipelines.

·Health economics of drug delivery are under scrutiny, as payers assess how delivery systems influence value, pricing pressure, and access for emerging therapies.

Partnering to Advance Drug Delivery Innovation

, director of strategic innovation for rare diseases at Chiesi USA (1). The session will explore critical considerations for evaluating external drug delivery platforms, with panelists offering perspectives from both early-stage and mature development environments.

, research & development advisor for Genetic Medicine at Eli Lilly and Company

, head of advanced drug delivery at AstraZeneca

, head of global biopharmaceutics and senior research fellow at Pfizer

, director of portfolio strategy & integration at Bristol-Myers Squibb.

The discussion will focus on partnership pathways, strategic fit assessments, and the evolving landscape of delivery technology collaborations (1).

Pharmaceutical leaders share internal approaches to innovation

Representatives from Novartis and AbbVie will offer insights into their respective organizations' approaches to sourcing and integrating drug delivery and enabling technologies (1). 

, global head of innovation at Novartis, and 

, senior director of landscaping & platform definition at AbbVie, will present as part of the conference’s 

These presentations are expected to provide transparency into current technology scouting efforts and collaborative models used to accelerate therapeutic development (1).

Economic considerations in drug delivery adoption

 will explore the intersection of innovation and reimbursement, with a focus on the pharmacoeconomics of delivery-enabled therapeutics (1). The panel will assess how delivery systems influence value perception, pricing pressures, and market access—especially in the context of drug classes such as incretins and nucleic acid-based therapies.

Dr. Gail Ryan, PharmD, formerly of Point32Health.

Technology tracks cover injectable, non-injectable, and genetic delivery

The 2025 agenda includes a comprehensive lineup of drug delivery technology presentations categorized into several themes (1):

injectable formulations and delivery platforms

self-injection devices, wearables, and connected delivery tools

oral, nasal, pulmonary, and alternative non-injectable systems

cell and gene therapy delivery approaches

innovations in excipients and combination product platforms

primary packaging solutions including syringes and containers.

Emerging topics in advanced therapeutic delivery

Sessions will also explore specialized approaches to delivery challenges in advanced modalities (1). Topics include:

brain delivery via formulation and physical mechanisms

novel vehicles for genetic medicine administration

tissue-specific targeting for nucleic acid therapeutics

manufacturing scale-up strategies for clinical and commercial readiness

sustainability in drug and device development.

Industry participation reflects broad engagement

The conference will feature speakers and presenters from across the global pharmaceutical ecosystem (1). Participating companies include major developers and technology providers working in small molecules, biologics, cell and gene therapies, and RNA-based drugs.

Participating organizations include: Amgen, Astellas Pharma, AstraZeneca, Beam Therapeutics, Biogen, Boehringer Ingelheim, Bristol-Myers Squibb, Chiesi USA, Eli Lilly and Company, Genentech, Gilead Sciences, GSK, Merck, Moderna, Novartis, Novo Nordisk, Pfizer, Regeneron, Roche, Sanofi, Sun Pharma, Takeda Pharmaceuticals, Teva Pharmaceuticals, UCB, and others.

PODD’s role in the drug delivery ecosystem

"PODD connects industry leaders and innovators who advance the design and development of drug delivery systems and devices to improve patient lives," said Andrew Goldstein, senior conference producer (2).

Designed to support scientific exchange and partnership building, PODD offers attendees a structured environment to explore delivery solutions for a wide range of therapeutic challenges (1). The forum supports deal-making, technology scouting, and collaboration-building across traditional boundaries in the pharmaceutical sector.

The full agenda release comes three weeks after keynotes were announced (3). At the time, Goldstein said, “In presenting a strategic-level program for R&D scientists and business development professionals, the PODD Conference is committed to securing keynotes who demonstrate excellence in science.”

Keynotes will feature prominent voices in pharmaceutical science and patient advocacy (3). 

, MD, PhD, co-founder and chief scientific officer of Regeneron, will join Massachusetts Institute of Technology’s 

, to discuss the current landscape and future potential of drug delivery innovation. 

, chief scientific advisor at Novo Nordisk, will present on the development of liraglutide and semaglutide, emphasizing how drug delivery intersected with the scientific and clinical advancement of these obesity treatments. Patient advocate 

 will share his experience living with sickle cell disease and undergoing treatment with a clustered regularly interspaced short palindromic repeats, or CRISPR, -based therapy, as well as his efforts to improve access to genetic medicines. 

, university distinguished professor at Northeastern University, will present on strategies for endogenous targeted delivery, addressing how biological transport mechanisms can be leveraged to improve drug efficacy and safety.



Partnership Opportunities in Drug Delivery 2025

Leading Drug Delivery Conference PODD Announces its 15th Annual Agenda

PODD Drug Delivery Conference Announces 2025 Keynotes. Press Release

The annual Boston conference will explore strategic partnering, device innovation, and delivery solutions for emerging therapeutics.

PODD 2025 Unveils Agenda Highlighting Innovation in Drug Delivery Technologies

ADELAIDE, SOUTH AUSTRALIA. - On November 15, 2018. – The University of South Australia is a public university, it is main campus is located on North Terrace in the Adelaide city centre | Image Credit: © arliftatoz2205 - stock.adobe.com

A novel once-weekly injectable formulation developed by researchers at the University of South Australia (UniSA) introduces a new drug delivery paradigm for Parkinson’s disease and potentially other chronic conditions (1). Designed to administer levodopa and carbidopa over seven days via a biodegradable, subcutaneous 

 forming implant, this system could reduce pill burden for patients while presenting significant implications across pharmaceutical discovery, development, and manufacturing (2).

“Our goal was to create a formulation that simplifies treatment, improves patient compliance, and maintains consistent therapeutic levels of medication,” said 

, professor of Pharmaceutical Science at the University of South Australia (1). “This weekly injection could be a game-changer for Parkinson’s care. Levodopa is the gold-standard therapy for Parkinson’s, but its short life span means it must be taken several times a day.”

 forming biodegradable implants enable sustained levodopa release, requiring novel aseptic processing and polymer-handling techniques.

· Formulation uses FDA-approved PLGA and Eudragit polymers, spotlighting the growing role of excipient science in lifecycle drug innovation.

· Weekly injectable supports 505(b)(2) and EMA hybrid pathways, offering a streamlined regulatory route for reformulated legacy therapeutics.

Extending innovation through delivery science

Although the active ingredients—levodopa and carbidopa—are well-established in Parkinson’s therapy, the innovation lies in the delivery system. This approach reflects a growing emphasis on lifecycle management of existing drugs through formulation science, an area increasingly recognized as critical in modern drug discovery pipelines (3). The use of a pH-responsive polymer blend—PLGA (an FDA-approved biodegradable polymer) and Eudragit L-100—to achieve consistent plasma levels over a full week demonstrates the role of excipient and polymer science in extending the therapeutic impact of legacy molecules (2).

This formulation trend aligns with industry strategies focused on enhancing the therapeutic index and patient adherence of known pharmacophores through novel delivery systems, especially in diseases with long treatment durations and high unmet needs (4).

Patient-centric reformulation and regulatory potential

From a drug development perspective, UniSA’s injectable system offers a reformulation strategy that aligns with patient-centric design principles, increasingly prioritized by regulators and industry sponsors alike (5). Parkinson’s disease currently requires multiple daily doses of oral levodopa/carbidopa to manage motor symptoms due to the drugs’ short plasma half-lives and rapid peripheral metabolism (6). This leads to fluctuations in drug levels and decreased clinical control, a challenge that this once-weekly delivery system aims to address by providing a more stable pharmacokinetic profile (2).

 data from UniSA’s study indicated that more than 90% of levodopa and 81% of carbidopa were released steadily over seven days (1). The biodegradable implant degraded by more than 80% during that period, and cell viability tests showed no significant cytotoxicity (2). These early findings support the potential for human translation.

Importantly, development teams may consider regulatory pathways, such as FDA’s 505(b)(2) route or the European Medicines Agency’s hybrid applications, which allow for the incorporation of published literature and data on approved actives, reducing the burden of full clinical development while still requiring robust evidence of safety and efficacy for the new delivery system (7,8).

 forming injectable implants introduces new challenges and considerations for pharmaceutical manufacturing. UniSA’s system requires formulation techniques that accommodate both the controlled encapsulation of active compounds and the precise modulation of polymer degradation kinetics. Manufacturing such systems typically involves aseptic processing, solvent handling, and quality control of biodegradable polymer blends, all of which differ significantly from those used in oral solid dosage forms (9).

Administering the formulation through a 22-gauge needle also implies the need for tight control over viscosity, injectability, and sterility during scale-up, especially under good manufacturing practice conditions (2). In addition, such polymers as PLGA and pH-sensitive materials like Eudragit may require controlled storage temperatures to maintain stability and prevent premature polymerization or phase separation (10). These requirements may necessitate cold-chain logistics infrastructure or temperature-monitored storage and shipping protocols.

Moreover, the potential for this delivery platform to be customized for release periods ranging from days to weeks presents a modular approach that could appeal to contract development and manufacturing organizations and large-scale manufacturers focused on flexible manufacturing lines for different release profiles and therapeutic indications (2).

Combination therapy and platform applications

UniSA researchers also noted that the injectable platform could be adapted for other chronic conditions requiring sustained drug exposure, including cancer, neurodegenerative diseases, diabetes, chronic pain, and infectious diseases (1). In this context, the platform offers potential for future combination products, in which two or more actives could be delivered together in a single depot, especially relevant in Parkinson’s, for which adjunctive therapies like MAO-B inhibitors or dopamine agonists are frequently prescribed alongside levodopa (6).

Such versatility could support combination regimens that improve therapeutic outcomes and simplify complex dosing schedules, a goal aligned with broader efforts in pharmaceutical development to enhance real-world adherence and reduce polypharmacy burden (11).

The UniSA injectable system for Parkinson’s therapy illustrates how innovative drug delivery mechanisms can drive significant therapeutic and operational advances, even without introducing new molecular entities. For professionals working across the drug development spectrum, this approach exemplifies how novel formulations can improve adherence, reduce dosing frequency, and create new opportunities for market differentiation.

As the researchers move toward human trials, this work may catalyze further investment in long-acting delivery systems that support sustained and controlled drug exposure. Downstream, it may also prompt regulatory and commercial stakeholders to reconsider value frameworks for reformulated legacy therapies with demonstrated real-world impact.


Weekly Injection Could be Life Changing for Parkinson’s Patients. Press Release

Nakmode, D.; Abdella, S.; Song, Y.; Garg, S. 

Development of an In-situ Forming Implant System for Levodopa and Carbidopa for the Treatment of Parkinson’s Disease

Effects of New Formulation Strategy on Life Cycle Management in the US Pharmaceutical Industry

Repurposing and Reformulation: Unlocking New Therapeutic Avenues through Formulation Optimization

Patient-Focused Drug Development: Collecting Comprehensive and Representative Input: Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders

 (CDER, CBER, June 2020) (accessed July 15, 2025).

Olanow, C.; Agid, Y.; Mizuno, Y.; et al. 

Levodopa in the Treatment of Parkinson’s Disease: Current Controversies

Applications Covered Under Section 505(b)(2)

 (CDER, October 1999) (accessed July 15, 2025).

. ema.europa.eu (accessed July 15, 2025).

The Manufacturing Techniques of Various Drug-loaded Biodegradable Microspheres

Poly Lactic-co-Glycolic Acid (PLGA) as Biodegradable Controlled Drug Delivery Carrier

Vrijens, B.; De Geest, S.; Hughes, D.; et al. 

A New Taxonomy for Describing and Defining Adherence to Medications

Biodegradable weekly implant delivers stable levodopa levels, aiming to streamline Parkinson’s care while reshaping drug reformulation and delivery strategies.

Why You Should Know About a New Weekly Injectable for Parkinson’s

Thornhill, Ontario, Canada - October 31, 2020: LEO Pharma Canada sign outside their head office building in Thornhill, Ontario, Canada. LEO Pharma AS is a multinational Danish pharmaceutical company | Image Credit: © JHVEPhoto - stock.adobe.com.

LEO Pharma has entered into a global licensing and transfer agreement with Boehringer Ingelheim for the commercialization and continued development of spesolimab, marketed as Spevigo (1). The monoclonal antibody (mAb) is the first targeted treatment approved for flares of generalized pustular psoriasis (GPP), a rare and potentially life-threatening inflammatory skin condition.

The agreement places LEO Pharma at the helm of Spevigo’s next phase, building on its presence in more than 40 countries—including the United States, Japan, China, and across Europe—where it is approved for the treatment of GPP flares in adults. Regulatory authorizations have also been granted for broader GPP indications in the US, European Union, and China (2).

Targeting an unmet need in rare dermatologic diseases

·LEO Pharma will lead global commercialization and development of Spevigo, expanding its role in rare dermatology drug manufacturing and access.

·The IL-36-targeting monoclonal antibody is advancing beyond GPP, with new development efforts focused on other rare autoinflammatory skin diseases.

·The agreement includes milestones and royalties, highlighting strategic deal-making trends in pharma for rare disease therapies with high unmet need.

Spesolimab is a humanized, selective mAb that blocks activation of the interleukin-36 (IL-36) receptor, a signaling pathway central to the development of several autoinflammatory skin diseases (3–5). In GPP, the IL-36 pathway is considered a critical driver of disease activity, making it a key target for therapeutic intervention.

GPP is a severe and unpredictable condition characterized by widespread sterile pustules, skin inflammation, and systemic symptoms such as fever, fatigue, and in some cases, organ failure (6–10). Patients may experience relapsing or persistent disease with varying intensity, and quality of life is often significantly impaired. Clinical studies show GPP is distinct from plaque psoriasis, with unique underlying pathology and treatment needs (6,7).

Spevigo has been evaluated in the largest clinical development program dedicated to GPP to date (1). It remains the only treatment for GPP flares that specifically targets IL-36 and has been validated in a randomized, placebo-controlled trial. Further investigation is underway to assess its potential in other IL-36-mediated dermatologic diseases with limited treatment options.

Under the agreement, LEO Pharma assumes responsibility for both global commercialization and further clinical development of the drug (1). The company plans to leverage its existing medical dermatology infrastructure to increase awareness and patient access to the treatment.

“Partnering to bring Spevigo to more patients is more than a strategic step—it means the opportunity to help people living with GPP by addressing a disease with limited treatment options and aiming to improve their quality of life,” said Christophe Bourdon, CEO of LEO Pharma (1). “We are inspired by Boehringer Ingelheim’s pioneering efforts and thrilled to build on that foundation.”

The deal is expected to close in the second half of 2025, pending regulatory clearance (1). Boehringer Ingelheim will receive an upfront payment of EUR 90 million, in addition to potential milestone payments and tiered royalties based on commercial performance.

Boehringer Ingelheim’s initial development of Spevigo marked a significant milestone for GPP patients and medical dermatology (1).

“We are immensely proud of what Spevigo represents for people living with GPP and bringing innovation to this community of patients has been a powerful reflection of our purpose to transform lives,” said Shashank Deshpande, chairman of the Board of Managing Directors and head of Human Pharma at Boehringer Ingelheim (1). “Spevigo holds a significant promise, and ensuring it reaches its full potential requires continued focus, and expertise in medical dermatology. With over six decades of singular dedication to this field, LEO Pharma is exceptionally well-positioned to build on the strong foundation we’ve laid.”

GPP remains a highly complex and underserved condition (1). Mortality rates for severe cases can range from 2% to 16%, often due to complications like multisystem organ failure and sepsis. Many patients also experience comorbidities that increase both personal and healthcare system burdens.

Boehringer Ingelheim and LEO Pharma Enter Partnership to Commercialize and Further develop Spevigo (Spesolimab)

European Commission Approves SPEVIGO for New and Expanded Indications in Generalized Pustular Psoriasis

Morita. A.; Strober, B.; Burden, A.D.; et al. 

Efficacy and Safety of Subcutaneous Spesolimab for the Prevention of Generalised Pustular Psoriasis Flares (Effisayil 2): An International, Multicentre, Randomised, Placebo-controlled Trial

Choon, S.; Lebwohl, M.; Marrakchi, S.; et al. 

Study Protocol of the Global Effisayil 1 Phase II, Multicentre, Randomised, Double-blind, Placebo-controlled Trial of Spesolimab in Patients with Generalized Pustular Psoriasis Presenting with an Acute Flare

Bachelez, H.; Choon, S.; Marrakchi, S.; et al. 

Trial of Spesolimab for Generalized Pustular Psoriasis

Pathophysiology of Generalized Pustular Psoriasis

Prinz, J.; Choon, S.; Griffiths, C.; et al. 

Prevalence, Comorbidities and Mortality of Generalized Pustular Psoriasis: A literature Review

Clinical Course and Characteristics of Generalized Pustular Psoriasis

Gooderham, M.; Van Voorhees, A.; Lebwohl, M. 

An Update on Generalized Pustular Psoriasis

Reisner, D.; Johnsson, F.; Kotowsky, N.; et al. 

Impact of Generalized Pustular Psoriasis from the Perspective of People Living with the Condition: Results of an Online Survey

The IL-36 inhibitor is being explored for additional inflammatory skin conditions, aiming to expand treatment options for underserved patient groups.

LEO Pharma Takes Over Global Development of Spevigo for Rare Skin Disease GPP

Silver Spring, MD, USA - June 25, 2022: The FDA White Oak Campus, headquarters of the United States Food and Drug Administration, a federal agency of the Department of Health and Human Services (HHS) | Image Credit: © Tada Images - stock.adobe.com

On July 10, 2025, FDA made public more than 200 previously confidential decision letters—known as Complete Response Letters (CRLs)—issued between 2020 and 2024 for drug and biologic applications that were not approved during their initial review cycle (1). These letters provide detailed reasons why an application could not be approved in its submitted form, including issues related to safety, efficacy, manufacturing deficiencies, and bioequivalence (1). "This action is a double-edged sword," said Biopharm International Editorial Advisory Board Member Steven Walfish, President of Statistical Outsourcing Services. "The transparency should provide consumers and the capital markets with long overdue information to why drugs applications did not meet the threshold for approval. This information will help the industry to understand the current thinking of the agency on certain aspects of the clinical and CMC process. On the other hand, it opens the FDA up to scrutiny that they are not applying the standards in an even-handed manner.”

Previously, the FDA had not routinely shared CRLs publicly. Sponsors often disclosed minimal information when announcing a non-approval. A 2015 cross-sectional study published in 

 found that drug developers omitted around 85% of safety and efficacy concerns noted by the FDA in their public statements (2). Moreover, when the FDA recommended a new clinical trial, that fact was left out of sponsor communications in approximately 40% of cases.

The newly released letters are available through the openFDA platform and were redacted to remove trade secrets or confidential commercial information (1). According to the agency, only CRLs tied to subsequently approved products were included in this initial batch. The FDA is reviewing its archives and has indicated it may release additional letters in the future as part of a broader transparency initiative (1,3).

FDA Commissioner Marty Makary, MD, MPH, stated that the move is intended to reduce the “guessing game” for drug developers and investors and help foster more predictable pathways for therapeutic approvals (1).

Critics of the previous system have long pointed to the lack of transparency as a factor in repeated regulatory missteps across the industry. The inability to learn from others’ failures has arguably contributed to inefficiencies in clinical development and strategic planning. The publication of CRLs is expected to give companies and investors deeper insight into the agency’s decision-making process and highlight common pitfalls across clinical, manufacturing, and regulatory domains (4).

However, some observers note that the release raises questions about consistency, timing, and scope. For example, it remains unclear whether future CRLs for non-approved applications will be published or whether sponsors will have input into the redaction process. Legal analysts have also flagged the importance of maintaining confidentiality protections while expanding access to regulatory history (5).

While reactions to the policy shift have been mixed, the publication of CRLs represents a notable change in how the FDA communicates its review decisions and could signal a broader evolution in U.S. regulatory transparency standards.

FDA Embraces Radical Transparency by Publishing Complete Response Letters

Describing the Content of FDA Complete Response Letters and How Sponsors Publicly Report Them: Cross Sectional Study

FDA Releases ‘Initial Batch’ of More Than 200 Drug Rejection Letters

FDA says the move boosts regulatory transparency, offering insights into common approval barriers in drug applications and reducing guesswork.

Featured Content

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

Behind the Headlines Episode 28

Ask the Expert

Drug Digest December 2025

A “Plan, Prevent, Prove” Approach