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

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Behind the Headlines Episode 28

SVP of Manufacturer, Quality, and Replenishment Operations, Cencora

The FDA finalized a rule standardizing the NDC to a uniform, 12-digit format

The views and opinions expressed herein are those of the author and do not necessarily reflect the views or positions of

The National Drug Code (NDC) is the FDAs standard for uniquely identifying drugs in the US, appearing everywhere from packaging and DEA records to pharmacy and financial systems. More than a label, it serves as the pharmaceutical industry’s primary transactional identifier across manufacturers, distributors, pharmacies, payers, 

The rule takes effect on March 7, 2033, giving stakeholders a seven-year preparation window, and an additional three-year transition period to update labeling, systems, and data infrastructure. While standardizing this drug information will improve consistency and interoperability across the supply chain, it is much more complex than a simple formatting change. This rule presents a Y2K-style challenge for pharma, requiring unprecedented alignment and a coordinated shift across the industry.

Currently, the NDC is a unique 10-digit number that appears in several different formats (4-2-2, 5-3-2, or 5-4-1) listing a labeler, product, and packaging code. The new 12-digit structure introduces a single, standardized format (6-4-2). Transitioning away from the current NDC format will create a uniform system across all products, while resolving the limitations of the current 5-digit labeler code structure.

However, the shift is much more complex than it seems. The current linear barcode can’t accommodate a 12-digit code, requiring a coordinated shift to a different barcode format, most likely that of a 2D data matrix. With the implementation of the 

 (DSCSA), which required the 2D data matrix for prescription drugs, the industry has already started to adopt these barcodes. The challenge now is extending them more broadly, particularly for over-the-counter drugs, where no standardized approach exists to shift to a 2D barcode.

Another nuance to consider is that during the three-year transition period from 2033 to 2036, FDA does not intend to object to the continued use of 10-digit NDCs on drug labels. FDA has also noted that the rule does not change NDC formats used for other purposes, including the HIPAA standard 11-digit format used for reimbursement, meaning systems may need to transact across multiple formats simultaneously.

While this NDC shift can be compared to a Y2K-style moment for the pharma industry, the key difference is that it’s not just up to individual stakeholders to update their own systems. This requires simultaneous, external coordination across the entire supply chain, and while systems won’t fail outright, there is a lot at stake. Errors can disrupt reimbursement, distribution, and even retail availability. Any system that touches an NDC must evolve its barcode scanners, point-of-sale systems, and inventory and dispensing systems, or the industry will risk widescale fragmentation.

The operational challenges for implementation can be separated into the two main drug streams: prescription drugs and over-the-counter drugs. For prescription drugs, there is a need for industry-wide technical specifications to ensure systems can transact across multiple NDC formats during the transition period. This raises a series of open questions: who defines this standard, how is it communicated, and who is leading the charge? Without clear alignment, even small inconsistencies can create downstream disruption.

On the other hand, over-the-counter drugs also present a major risk area. The FDA has indicated that linear or nonlinear barcode formats may be used to encode the NDC if they conform to recognized standards, and that a 2D data matrix barcode can encode the 12-digit NDC for products subject to DSCSA requirements. Even so, broader implementation across over-the-counter products and non-pharmacy retail environments may add complexity for stakeholders that need to update scanning, labeling, and data systems.

There are seven years before this rule takes effect. While this may seem like a long time, the scale of this shift leaves little room for delay. The pharma industry must act now to effectively coordinate in time. Without early alignment across entities, the industry risks fragmentation and the kind of disruption many feared during Y2K.

For manufacturers, preparation begins with a clear understanding of impact. This starts with conducting a comprehensive impact assessment of SKUs, labeling formats, and downstream system dependencies. The larger the portfolio of SKUs, the more time it will take to inventory. From there, organizations can begin planning for updates, which is where the true challenge begins.

The industry has already demonstrated its ability to align on major operational changes through DSCSA, where wholesalers led the charge for manufacturers and dispensers toward the implementation of serialized drug tracking and standardized barcodes. The key difference with NDC is that this transition introduces a broader, more fragmented set of stakeholders

Success will hinge on aligning standardization and leadership efforts.

Overall, this must be treated as more than a labeling shift or compliance exercise to avoid any operational and commercial disruptions or worse, a Y2K-moment for pharma. To effectively bridge the gap between legacy 10-digit data and the new 12-digit standard, this must be approached as a full-scale system transformation, with widescale coordination across the entire supply chain.

US Food and Drug Administration. National Drug Code Format

https://www.fda.gov/drugs/electronic-drug-registration-and-listing-system-edrls/national-drug-code-format

Food and Drug Administration. Revising the National Drug Code Format and Drug Label Barcode Requirements

https://www.federalregister.gov/documents/2026/03/05/2026-04368/revising-the-national-drug-code-format-and-drug-label-barcode-requirements

Articles

FDA finalized a uniform 12-digit National Drug Code (NDC) (6-4-2) to replace today’s variable 10-digit formats, effective March 7, 2033, followed by a 2033–2036 transition. The change extends well beyond relabeling, forcing barcode evolution because linear codes cannot accommodate 12 digits, accelerating reliance on 2D data matrix—especially challenging in OTC and non-pharmacy retail. Concurrent coexistence of 10-, 11- (HIPAA), and 12-digit formats heightens interoperability risk, demanding supply-chain-wide technical alignment to avoid reimbursement, distribution, and availability disruptions.

FDA's 12-digit NDC rule demands industry-wide barcode, labeling, and data system overhauls, manufacturers must act now to avoid pharma's Y2K moment.

Preparing for Pharma’s “Y2K Moment”: What the Shift to a 12-Digit NDC Means for Manufacturers

In regulatory news in June 2026, the US FDA accepted the first in silico drug development tool as part of its ISTAND program, approved a variety of drugs that included treatments for rare conditions, and issued guidance on the acceleration of cell and gene therapy.

On June 3, 2026, the agency accepted a letter of intent for an 

 into its Innovative Science and Technology Approaches for New Drugs (ISTAND) DDT Qualification Program.

 The DDT is an artificial intelligence-driven digital liver model that predicts drug induced liver injury and will help reduce the use of animals in a nonclinical space and improve predictive toxicology in humans.

 posted June 8, Dr. Iilun Murphy, director Office of Generic Drugs, Center for Drug Evaluation and Research (CDER), and Dr. Mike Kopcha, director Office of Pharmaceutical Quality, CDER, provided insight into the agency’s approach to generic drug oversight.

 Murphy and Kocha acknowledged that the manufacturing of generic drugs is complex and encouraged manufacturers to adopt mature quality management practices beyond what is required by current good manufacturing practices. They also restated the FDA’s support for onshore manufacturing.

over-the-counter (OTC) intranasal naloxone product

, Rextovy, was approved by the FDA on June 16, 2026 for the emergency treatment of opioid overdose.

 The action is part of FDA’s priority to reduce opioid deaths by broadening access to these types of treatments that rapidly reverse the effects of opioid overdose.

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June saw regulators look back at their achievements, take stock of drug shortages, and advance treatments for rare diseases.

Regulatory Actions for June 2026

BioPhorum formed a group aimed at accelerating innovation across small molecule manufacturing

BioPhorum, a global forum for biopharmaceutical industry collaboration, has formed a new working group aimed at accelerating innovation across small molecule manufacturing.

 The Synthetic Molecule Process Innovation Phorum brings together 20 organizations spanning global pharmaceutical companies, specialist CDMOs, technology providers, and regulatory bodies, with the goal of aligning the broader ecosystem around faster, more coordinated adoption of new manufacturing approaches.

Participants include major pharmaceutical manufacturers alongside regulatory and standards bodies, such as the UK's Medicines and Healthcare products Regulatory Agency, the British Pharmacopoeia, and the US Pharmacopeia.

 Their involvement signals an intent to make the group a direct channel between industry practice and regulatory expectations, rather than a forum limited to internal industry discussion.

Why Is Small Molecule Manufacturing Under Pressure?

The initiative responds to a shift already underway in drug development pipelines.

 Small molecule therapies, once associated largely with niche or high-potency compounds, are increasingly central to high-volume treatments in oncology, neurology, obesity, and immunology. That shift is placing new demands on manufacturing infrastructure, which must scale efficiently while maintaining supply chain resilience. “As pipelines fill with complex small molecules, from next‑generation obesity treatments to breakthrough therapies in oncology and immunology, the question is no longer whether science can deliver, but whether manufacturing can keep up,” stated Andrea Johnston, senior director, BioPhorum, in a press release.

 “By bringing global pharma and specialist CDMOs around one table, the BioPhorum Synthetic Molecule Process Innovation is designed to turn market momentum into robust, efficient, and sustainable ways of making these products at scale.”

This framing matters because it reflects a broader industry pattern: therapeutic innovation is outrunning the operational systems meant to support it.

 Facilities built around older production assumptions may struggle to accommodate the complexity and scale of newer synthetic molecule pipelines without coordinated updates to process design, regulatory guidance, and data infrastructure.

Following a two-day planning session in Cork, Ireland, the collaboration identified four initial focus areas.

 The first addresses peptide manufacturing, specifically capacity constraints and raw material availability, which have become recurring bottlenecks as peptide-based therapies expand. The second targets regulatory alignment, including global acceptance of solvent reuse practices and clearer chemistry, manufacturing, and controls guidance for synthetic peptide production under biologics license applications. The third focuses on model-based predictive control, aiming to develop frameworks for robust, end-to-end control strategies across manufacturing processes. The fourth centers on knowledge lifecycle management, an effort to standardize how data are shared and interpreted across a product's development and manufacturing lifecycle.

Each of these areas touches a persistent operational challenge in the sector: fragmented regulatory expectations across regions, inconsistent data practices between organizations, and supply constraints that can delay production timelines even when clinical development proceeds smoothly.

 A coordinated, cross-industry approach to these issues could reduce duplicated effort among individual companies working through similar problems independently.

The launch also reflects a wider consolidation trend among industry organizations that support manufacturing best practices, as groups expand their scope to address adjacent operational challenges rather than operating within narrower technical silos.

The formation of this group is worth monitoring less for what it announces today than for what it may produce over time: potential shifts in regulatory guidance, emerging standards for predictive control systems, and clearer expectations around peptide manufacturing that could influence facility planning and compliance strategy in the coming years.

What Does Market Growth Show About the Urgency Behind This Collaboration?

Recent figures reinforce the timing of BioPhorum's initiative.

 The global small molecule API contract development and manufacturing market was valued at $46.6 billion in 2025 and is projected to grow to $80.7 billion by 2033, expanding at a compound annual rate of 7.3%. Analysts attribute this growth partly to rising demand for complex APIs, supply chain resilience initiatives amid shifting geopolitics, and increasing digitalization and regulatory evolution. Phorum's four work streams aim to address these same pressures collectively rather than company by company.

BioPhorum. BioPhorum unites regulators, big pharma and CDMOs to accelerate synthetic molecule innovation. Press release. July 1, 2026.

Grand View Research. Small molecule CDMO market size | industry report, 2026. July 1, 2026. 

https://www.grandviewresearch.com/industry-analysis/small-molecule-cdmo-market-report

BioPhorum has launched the Synthetic Molecule Process Innovation Phorum to coordinate faster adoption of advanced small-molecule manufacturing practices across 20 stakeholders, including large pharma, CDMOs, technology vendors, and MHRA/BP/USP. The effort reflects mounting scale and complexity demands from oncology, immunology, neurology, and obesity pipelines, where operational capability increasingly constrains product delivery. Initial workstreams target peptide capacity and raw-material bottlenecks, global regulatory alignment (eg, solvent reuse and CMC for peptides under BLAs), model-based predictive control, and standardized knowledge lifecycle management.

BioPhorum launches a 20-member collaboration uniting pharma, CDMOs, and regulators to address manufacturing challenges in small molecule drug production.

BioPhorum Unites 20 Organizations to Modernize Small Molecule Production

Blood drop, floating stem cells. Hematopoietic stem cell collection, regenerative medicine, blood-derived stem cells, donor therapy, transplantation concept | Image Credit: ©Елена Бутусова -stock.adobe.com

The FDA announced on June 30, 2026 that it has approved Tregzi for the treatment of chronic graft-versus-host disease (GVHD), a condition in which transplanted donor blood cells attack the recipient's body.

 Tregzi, a donor-derived cellular immunotherapy developed by Orca Biosystems, is the first regulatory T-cell-based immunotherapy approved for improving chronic GVHD-free survival in this patient population, marking a shift in how transplant centers might approach an outcome that has historically been difficult to prevent through pharmacologic means alone.

"For patients with blood cancers who need stem cell transplantation, chronic graft-versus-host disease has long been one of the most feared and difficult-to-prevent complications," said Karim Mikhail, B.Pharm., M.S., acting director of the Center for Biologics Evaluation and Research, in a press release.

 "Today's approval offers a genuine new approach that can help reconstitute the immune system while substantially reducing that risk and reflects the promise of what cellular therapy can deliver for patients."

“For transplant physicians, one of our greatest challenges has long been preserving the vital graft-versus-leukemia effect while minimizing the risk of GVHD and infection,” said Miguel-Angel Perales, M.D., medical oncologist and chief of the Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center, in a press release.

 “The FDA approval of TREGZI signals a new era in transplant medicine. This precision-engineered cell therapy is built on the foundational principles established by our early CD34 cell selection work and can now be delivered at scale, equipping providers with a new option to reduce serious toxicities and improve treatment outcomes."

How the Cell Therapy Is Manufactured and Administered

Unlike a standard stem cell transplant, which typically infuses a single population of donor cells, Tregzi is composed of 3 distinct cell components manufactured from a single donor collection: purified hematopoietic stem and progenitor cells, regulatory T cells, and conventional T cells. All 3 are derived from the mobilized peripheral blood of a donor who is an 8/8 human leukocyte antigen match, either a relative or an unrelated donor.

Regulatory T cells are immune cells that help regulate immune responses and maintain immune tolerance. By isolating and formulating this cell type alongside the stem and progenitor cells and conventional T cells, the therapy is intended to support reconstitution of a patient's blood-forming and immune systems while lowering the likelihood that donor-derived cells will attack healthy tissue. Patients receive the treatment after a course of chemotherapy that prepares the body for the transplant.

"We founded Orca Bio on the audacious goal to engineer living cells into curative medicines, rooted in the belief that single-cell precision could fundamentally rewrite patient outcomes," said Nate Fernhoff, PhD, co-founder and chief executive officer of Orca Bio, in a press release.

 "The FDA approval of TREGZI is a significant milestone that stands on the shoulders of decades of pioneering science. As we enter this next chapter, our focus turns to the immense responsibility of delivering TREGZI reliably, precisely and safely to the patients and families counting on us."

Trial Data Behind the Chronic GVHD-Free Survival Endpoint

The approval was based on findings from PRECISION-T, a randomized controlled trial that enrolled 187 adult patients with blood cancers, including acute leukemia and myelodysplastic syndrome. Participants were assigned to receive either Tregzi or a standard stem cell transplant, and the trial's primary endpoint was chronic GVHD-free survival. This was measured as the time from transplantation to the earliest occurrence of either death from any cause or the first onset of moderate or severe chronic GVHD, tracked within 2 years of the transplant date.

After 1 year, 78% of patients who received Tregzi met this endpoint, compared with 38.4% of those who received a standard transplant. When death was accounted for as a competing risk, 12.6% of Tregzi recipients developed serious chronic GVHD within a year, versus 44% among patients who received a standard transplant. According to FDA, the trial's results demonstrate clinical benefit for the studied population; therefore, the agency concluded that Tregzi's benefits outweigh its risks.

The safety profile observed in the trial was consistent with patients undergoing stem cell transplantation, with infection as the most common adverse event. No  severe reactions occurred during infusion, and no cases of graft failure were reported during the study period. 

“Developing this concept from early foundational research in our labs based upon the fundamental biology of regulatory T cells, to it now receiving the first FDA approval for a therapy that utilizes highly purified Tregs, is a defining moment for the transplant community," said Robert Negrin, M.D., professor of medicine, blood and marrow transplantation at Stanford Medicine, in a press release.

 "The peer-reviewed findings demonstrated this precision-engineered cell therapy delivered improved GVHD-free survival alongside less toxicity, including fewer serious infections and lower non-relapse mortality."

The application received both Orphan Drug and Regenerative Medicine Advanced Therapy designations ahead of its approval.

FDA approves new treatment that uses donor immune cells to prevent serious complications in blood cancer patients. Press release. FDA. June 30, 2026. 

https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-uses-donor-immune-cells-prevent-serious-complications-blood-cancer

Orca Bio’s TREGZI™ receives U.S. FDA approval as first and only precision-engineered cell therapy for allogeneic transplant in adults with hematological malignancies. Press release. Orca Bio. June 30, 2026. 

https://orcabio.com/orca-bios-tregzi-receives-u-s-fda-approval-as-first-and-only-precision-engineered-cell-therapy-for-allogeneic-transplant-in-adults-with-hematological-malignanciesorca-bio-adds-east/

FDA approval of Tregzi introduces the first regulatory T-cell–based donor-derived cellular immunotherapy to improve chronic GVHD-free survival after allogeneic transplant in adults with hematologic malignancies. The product is manufactured from a single 8/8 HLA-matched donor mobilized peripheral blood collection into three components—purified HSPCs, regulatory T cells, and conventional T cells—administered following conditioning chemotherapy. In the randomized PRECISION-T trial, Tregzi markedly reduced moderate-to-severe chronic GVHD and improved the composite survival endpoint, with a transplant-consistent safety profile.

FDA has approved Tregzi, the first regulatory T-cell therapy shown to reduce chronic GVHD and improve survival in blood cancer transplant patients.

FDA Grants First-in-Class Approval for Treg Cell Therapy in Blood Cancer Transplant Patients

Macro closeup of young woman face portrait in profile side with Grave's disease hyperthyroidism symptoms of ophthalmopathy bulging eyes proptosis edema | Image Source: ©Andriy Blokhin -stock.adobe.com

Roche announced on June 30, 2026 that FDA granted priority review to the company’s supplemental biologics license application (sBLA) for satralizumab (Enspryng) for the treatment of thyroid eye disease (TED).

The agency accepted the filing after 2 randomized, placebo-controlled global phase 3 SatraGO studies found consistent improvements of TED symptoms and a more favorable safety profile when compared with currently available treatments. The study data were presented at the American Society of Ophthalmic Plastic and Reconstructive Surgery in October 2025.

“The FDA’s decision to grant priority review to Enspryng is an important step toward expanding treatment options for people living with thyroid eye disease,” said Levi Garraway, MD, PhD, Roche’s chief medical officer and head of Global Product Development, in a press release.

 “By targeting the underlying disease biology with a novel mechanism of action, this subcutaneous therapy has the potential to introduce a new treatment approach that combines clinical efficacy and a favorable safety profile with the convenience of at-home administration.”

TED is progressive, rare inflammatory autoimmune disease that can threaten sight. Symptoms include redness, swelling, eyelid retraction, eye bulging, double vision, and pain. TED impacts approximately 155 out of every 100,000 people and commonly occurs in people with hyperthyroidism.

Satralizumab is a humanized monoclonal antibody that targets IL-6, which is a messenger in the body’s inflammatory response, receptor activity, according to Roche. It was created using novel recycling antibody technology that allows for sustained IL-6 inhibition by binding to the IL-6 receptor.

Satralizumab is currently approved for the treatment of neuromyelitis optica spectrum disorder in 90 countries, including the United States and the European Union, where it has orphan drug designation. Roche is developing the treatment for additional neurological autoimmune and inflammatory diseases, including autoimmune encephalitis and myelin oligodendrocyte glycoprotein antibody-associated disease.

Roche Launches AXELIOS 1 Sequencing Platform

In other company news, on June 29, 2026, Roche launched its next-generation single-molecule sequencing platform, AXELIOS 1, powered by sequencing by expansion (SBX) technology. “The platform converts DNA/RNA into high signal‑to‑noise 'Xpandomers,' which are measured by a sequencing instrument using a reusable complementary metal–oxide–semiconductor sensor containing millions of nanopores. This architecture enables accurate, ultra‑rapid, flexible and high‑throughput, single‑molecule sequencing with near real‑time analysis,” the company stated in a press release.

“AXELIOS 1 will deliver a disruptive sequencing solution that combines high accuracy with unprecedented speed and scalability. These attributes, combined with our high level of cost efficiency, will enable the sequencing community to develop applications that previously were not feasible,” said Matt Sause, CEO of Roche Diagnostics, in a press release.

 “Additionally, in the future, AXELIOS 1 has the same potential to enable the next generation of clinical applications and unlock new frontiers in personalized healthcare.”

The new platform can be used for smaller, faster batches or larger projects and can produce whole-genome results in the same day. The flexible open setup gives researchers freedom to explore and create new workflows to gain knowledge of complex diseases.

Library prep kits and free open-source bioinformatics analysis suite will be offered by Roche. The platform can also be built for use of other life sciences tools.

FDA grants Priority Review to Roche’s Enspryng, the first and only at-home subcutaneous treatment option for thyroid eye disease (TED). Press release. Roche. June 30, 2026. 

https://www.roche.com/media/releases/med-cor-2026-06-30

Briceño C, et al. Primary Results of the Phase 3 SatraGO-1 and SatraGO-2 Trials: Efficacy and Safety of Satralizumab in Thyroid Eye Disease. Presented at the American Society of Ophthalmic Plastic and Reconstructive Surgery (ASOPRS) Fall Scientific Symposium, October 16–17, 2025.

Roche announces the launch of AXELIOS 1, a transformative next-generation sequencing platform. Press release. Roche. June 29, 2026. 

https://www.roche.com/media/releases/med-cor-2026-06-29

FDA granted priority review to Roche’s sBLA for satralizumab (Enspryng) in thyroid eye disease, supported by two global randomized phase 3 SatraGO trials showing consistent symptom improvement and a comparatively favorable safety profile. The subcutaneous, potentially at-home regimen targets IL‑6 receptor signaling via recycling antibody technology to sustain inhibition. Satralizumab is already approved for neuromyelitis optica spectrum disorder in ~90 countries. Roche also launched AXELIOS 1, a single-molecule sequencing system using sequencing-by-expansion for rapid same-day whole genomes, with an FDA action date expected within months.

The company’s thyroid eye disease treatment will be the first at-home subcutaneous disease-modifying treatment if approved.

Roche Gets Priority Review for Enspryng; Announces Sequencing Platform

Susan J. Schniepp is a fellow at Regulatory Compliance Associates, Inc. and a member of Pharmaceutical Technology's Editorial Advisory Board.

Siegfried Schmitt, PhD, is Vice President Technical at Parexel International, Siegfried.Schmitt@parexel.com.

Ask the Expert: Regulatory Developments Impacting Biologics

, Siegfried Schmitt, vice president, Technical, at Parexel, and Susan Schniepp, distinguished fellow at Nelson Labs, discuss regulatory and compendial announcements affecting biologics manufacturers.

The conversation was prompted by a question a PharmTech reader seeking to understand the significance of new publications from the FDA, the US Pharmacopeia (USP), and European regulators. Schniepp notes that these regulatory announcements are arriving in close succession, which is notable and appears to reflect a coordinated effort to support the rapidly evolving biologics field.

 have been released without accompanying specifications.

Schniepp views this as a deliberate move to assist manufacturers by providing validated methods while leaving specification-setting to individual companies based on their specific product. This indicates USP is helping streamline chemistry, manufacturing, and controls (CMC) sections in regulatory filings without imposing a one-size-fits-all standard.

, which was agreed upon in 2025, focuses on reducing drug shortages and eliminating supply chain bottlenecks.

 Schniepp highlights the concern that some critical raw materials or components used in biologics may be available from only a single supplier. If that supplier experiences a disruption, it can affect the entire industry. The act aims to address these single-source vulnerabilities and strengthen supply chain resilience.

issued a draft guidance on leveraging prior knowledge

 in the development of human gene therapy products incorporating genome editing.

 Rather than requiring each company to build a completely independent platform from scratch, the guidance would allow manufacturers to reference previously published studies and data to support their CMC filings, which could be a significant potential time-saver for an area where knowledge is often concentrated among a small number of firms. Both speakers emphasize that this guidance is still in draft form, with a public comment deadline of September 1, 2026, and encourage manufacturers to submit their perspectives.

How Are Regulators Supporting Biologics Development?

Schmitt broadens the conversation to the underlying intent behind these developments. Biologics, he notes, are frequently life-saving treatments that regulators are motivated to help bring to market. The challenge is not just regulatory approval, but ensuring these therapies can actually reach patients. That involves rethinking supply chains and manufacturing models. For cell and gene therapies, in particular, traditional centralized manufacturing and long-distance shipping are often not viable options. Both experts point to distributed, decentralized manufacturing (i.e., producing therapies physically closer to patients) as a likely direction for the industry.

Schniepp states that, in some cases, the final steps of manufacturing may need to occur in a clinical or hospital setting, such as combining a lyophilized product with water for injection. This requires close coordination between manufacturers and healthcare providers and must be factored into regulatory planning.

Rather than viewing regulators as lagging behind innovation or creating unnecessary burdens, the FDA, EMA, and USP are trying to keep pace with a fast-moving field and actively supporting industry progress, according to Schmitt and Schniepp, which is a positive development for patients, companies, and the broader ecosystem alike.

Biologics manufacturers should pay close attention to these announcements and engage with the process by submitting comments to both the FDA on its draft guidance and to USP on its proposed methodologies. Regulatory input from industry practitioners helps shape practical, workable standards.

Click on the video above to watch the experts answer the question:

“My company manufactures biologics, and we saw some recent actions by FDA, USP, and the EU, how do these regulatory actions impact us as a biologics manufacturer?”

USP publishes product-specific emerging biologics standards for public comment. USP. Press release. June 1, 2026. 

https://www.usp.org/news/usp-publishes-product-specific-emerging-biologics-standards-for-public-comment

EMA. EMA welcomes political agreement on the Critical Medicines Act

. News release. ec.europa.eu. 12 May 2026. https://ec.europa.eu/commission/presscorner/detail/en/ip_26_1017

https://health.ec.europa.eu/publications/proposal-critical-medicines-act_en

FDA issues draft guidance to help accelerate cell and gene therapies for patients. FDA. Press release. June 2, 2026. 

https://www.fda.gov/news-events/press-announcements/fda-issues-draft-guidance-help-accelerate-cell-and-gene-therapies-patients

Susan J. Schniepp is distinguished fellow at Nelson Labs and a member of 

Siegfried Schmitt is vice president, Technical at Parexel and a member of 

This episode of Ask the Expert takes a look at recent regulatory actions impacting biologics manufacturers.

Regulatory Developments Impacting Biologics

Oxylanthanum carbonate was rejected for the 2nd time by the FDA

Oxylanthanum carbonate, an investigational phosphate binder developed by Unicycive Therapeutics for hyperphosphatemia in patients with chronic kidney disease on dialysis, has received a second Complete Response Letter (CRL) from the FDA.

 The agency raised no concerns about clinical efficacy or safety and requested no additional data. Instead, the rejection rests entirely on unresolved compliance deficiencies at the third-party manufacturing vendor responsible for producing the drug substance, the same deficiencies cited in the original CRL issued in June 2025.

Between the two decisions, the company had pursued a now-familiar regulatory sequence: a Type A meeting with the agency in September 2025, an EU inspection of the same vendor that found no deficiencies, and a resubmission of the New Drug Application built on the vendor's reported progress toward inspection readiness.

 Despite that progress, the FDA had not completed its own inspection of the facility by the time of the second decision, leaving the compliance question unresolved and the application unapprovable on chemistry, manufacturing, and controls grounds alone.

What Types of Deficiencies Trigger CRL for a Third-Party Manufacturer?

CRLs tied solely to chemistry, manufacturing, and controls (CMC issues generally stem from facility-level compliance findings rather than problems with the drug product itself.

 Common triggers include unresolved findings from a pre-approval inspection, data integrity concerns, deviations from current good manufacturing practice, or failure to demonstrate inspection readiness within the review cycle. In this case, the company has stated that the citations at the vendor were not specific to oxylanthanum carbonate production, underscoring a recurring theme in CMC rejections: a facility's broader compliance status can stall an otherwise clean application, independent of how the investigational product itself performs. This distinction shifts the resolution pathway away from additional clinical work and toward facility remediation, an inspection, and confirmation of compliance, none of which the sponsor directly controls.

How Should Sponsors Avoid Resubmission Delays?

The case illustrates the value and the limits of contingency planning around contract manufacturing.

 The company identified and qualified a second manufacturing vendor that had already produced drug product following the first rejection, yet the resubmission still relied on the original vendor's compliance status, and the second rejection again hinged on that same facility. The lesson for sponsors managing third-party manufacturing relationships is that qualifying a backup vendor is necessary but not sufficient; the qualification timeline, validation batches, and any required inspections need to run far enough ahead of a Prescription Drug User Fee Act target date that switching vendors does not itself become the source of delay. Sponsors that wait until a CRL arrives to activate a secondary supply path risk repeating the same compliance dependency that triggered the original rejection, since regulatory timelines for vendor transitions rarely compress to match a resubmission cycle already in progress.

What Does This Mean for Novel Oral Solid Dosage Forms?

Oxylanthanum carbonate is formulated using proprietary nanoparticle technology intended to increase phosphate-binding potency and reduce pill burden compared with existing binders, a formulation approach gaining traction across oral solid dosage development as sponsors seek to improve adherence in chronic disease populations.

 This case is a reminder that novel particle-engineering platforms often depend on specialized contract manufacturers with the equipment and process expertise to produce them at scale, narrowing the pool of qualified vendors and concentrating regulatory risk at the facility level. As more oral solid dosage candidates rely on nanoparticle-based drug delivery, sponsors and their manufacturing partners should expect facility compliance history and inspection readiness to weigh as heavily on approval timelines as the underlying formulation science, particularly when specialized production capacity is limited and difficult to replicate quickly across alternate sites.

Reuters. US FDA declines to approve Unicycive’s drug for dialysis patients. Press Release. June 30, 2026. 

https://www.reuters.com/legal/litigation/us-fda-declines-approve-unicycives-drug-dialysis-patients-2026-06-30/

Unicycive Therapeutics, Inc. Unicycive Therapeutics Receives Complete Response Letter from FDA Regarding Resubmitted Oxylanthanum Carbonate (OLC) New Drug Application (NDA). Press Release. June 30, 2026. 

https://ir.unicycive.com/news/detail/123/unicycive-therapeutics-receives-complete-response-letter

Oxylanthanum carbonate, Unicycive’s investigational phosphate binder for dialysis-dependent CKD, received a second FDA Complete Response Letter driven solely by third-party manufacturing compliance deficiencies. No new clinical efficacy or safety issues were raised, and no additional data were requested. Despite a Type A meeting, an EU inspection finding no deficiencies, and an NDA resubmission citing vendor remediation progress, FDA had not completed its own facility inspection, leaving CMC approvability unresolved. The situation highlights how facility-level GMP status can dominate timelines for specialized nanoparticle-enabled oral solids.

The FDA again rejected Unicycive's oxylanthanum carbonate over third-party manufacturing compliance, not safety, spotlighting CMC risk in nanoparticle drug development.

Manufacturing Deficiencies Behind FDA's Oxylanthanum Carbonate Rejection

Antibody drug conjugate in blue with four drug compounds linked to IgG immunoglobulin; ADC in blue against blue background 3d render | Image Credit:  ©huenstructurebio.com -stock.adobe.com

Contract development and manufacturing organization (CDMO) Lonza announced on June 30, 2026 that it is enhancing its drug-linker center of excellence and expanding payload-linker manufacturing capacity at its Visp, Switzerland site.

 The company plans to add new commercial-scale capabilities to manufacture highly complex and highly potent APIs (HPAPIs) as well as antibody drug conjugate (ADC) payload linkers.

The expansion will add new manufacturing capacity within the company’s existing good manufacturing practice facility and add payload-linker production and purification to its dedicated analytical and process development laboratories. The facility can produce a range of payload-linker molecules and allows for multipurpose use and scale-out, with the ability to add additional suites for future demand.

The company’s ADC capabilities at the Visp site include the manufacturing of monoclonal antibodies, conjugation and drug product manufacturing, and quality control for handling highly potent payload-linker molecules. The expanded facility is planned for operation in 2028 and will support clinical and commercial programs.

Earlier investments in the Visp site included bioconjugation capabilities with the addition of 2 multipurpose 1200L manufacturing suites in 2024. The suites doubled the company’s multipurpose capacity. “We continue to see strong growth in the bioconjugates space as ADCs and other bioconjugated drugs increasingly progress towards commercialization,” Christian Morello, vice president, head of Bioconjugates, Lonza, said in a press release at the time.

 “This investment in our multipurpose commercial bioconjugation capacity addresses the growing market demand, enables us to support the growth of our customers and offers a flexible and integrated service for manufacturing bioconjugates.”

The growth of ADCs has increased demand for complex payload-linkers, according to Lonza, and these require customized synthesis and advanced purification technologies. The structural complexity of the molecules is also driving the need for the manufacture of specialized highly potent materials.

“The rapid growth of the ADC market is driving increasing demand for highly complex payload-linkers that require advanced manufacturing capabilities,” Christian Seufert, head of Advanced Synthesis at Lonza, said in a press release.

 “Lonza remains committed to investing and further developing its integrated ADC offering. This investment reflects that commitment by expanding our capacity to support this next generation of therapies and enabling our customers to progress efficiently from development to commercial supply, ultimately helping bring targeted, potentially life-changing treatments to patients.”

The HPAPI market is expected to expand to more than $60 billion by 2034.

According to Ester Masllorens, senior vice president of global R&D with Olon, “The HPAPI landscape is rapidly evolving, driven by breakthroughs in oncology, rare diseases, and advanced delivery platforms such as ADCs and peptides. The growing complexity and potency of these molecules requires sophisticated containment capabilities and manufacturing strategies.”

The challenges presented by the development and manufacturing of HPAPIs require specialized expertise, according to Masllorens, and those challenges and complexities continue to increase.

They require robust containment, validated cleaning procedures, strict protections for operators, as well as highly specialized synthesis and purification.

“To address the complexity of HPAPI development, the industry is increasingly adopting modular containment systems, green chemistry approaches, and digitalization,” stated Masllorens.

Lonza expands HPAPI capacity in Visp, further supporting its position in payload-linker manufacturing. Press release. Lonza. June 30, 2026. 

https://www.lonza.com/news/2026-06-30-07-00

Lonza to expand bioconjugation in Visp (CH) with two additional manufacturing suites. Press release. Lonza. November 12, 2024. 

https://www.lonza.com/news/2024-11-12-07-00

Grand View Research. High Potency Active Pharmaceutical Ingredients Market–By Product, By Drug Type, By Manufacturer Type, By Application–Global Forecast, 2025–2034. February 2025. 

https://www.gminsights.com/industry-analysis/high-potency-active-pharmaceutical-ingredients-market

Challener C. Trends and challenges impacting highly potent API development. 

https://www.pharmtech.com/view/trends-and-challenges-impacting-highly-potent-api-development

Lonza is expanding payload-linker and HPAPI manufacturing at its Visp, Switzerland site by upgrading its drug-linker center of excellence and adding commercial-scale capabilities within an existing GMP facility. The buildout integrates payload-linker production and purification into dedicated analytical and process development labs, enabling multipurpose, scale-out manufacturing with room for additional suites. The site already offers end-to-end ADC services spanning mAb manufacturing, conjugation, drug product, and QC for highly potent materials. Operations are targeted for 2028, supporting clinical-to-commercial supply.

The outsourcing company is adding commercial-scale capabilities to manufacture HPAPI and ADC payload-linkers.

Lonza Increases HPAPI Production at Visp Facility

FDA named seven participants for the February 2026 PreCheck Pilot Program to accelerate US-based pharmaceutical manufacturing and bolster supply-chain resilience. Selected firms span sterile small molecules, APIs, large-scale cell culture biologics, and cell/gene therapies, with sites in NY, NJ, IN, and NC. Applicants had to propose new domestic capacity addressing supply needs or unmet medical needs and commit to filings relying on the facility. The program uses pre-operational technical guidance followed by enhanced, facility-focused engagement to expedite evaluation and inspections.

The companies have been selected to participate in the pilot program to expand domestic pharmaceutical manufacturing and strengthen the US drug supply chain.