Regulatory Roundup—September 2026

In US regulatory news, the FDA opened applications for its Expedited IND Pilot on September 15.¹ The program pairs drug sponsors with qualified research institutions (QRIs), which support investigational new drug (IND) application preparation. Applications are due October 30, and the FDA expects to select 8–10 sponsor–QRI pairs for the first cohort. The design allows rolling review of individual IND components during the pre-IND phase rather than waiting for a complete package, with the aim of cutting clinical holds and shortening a drug-to-first-dose timeline that can run up to 2 years in the United States.

Dr. Lisheng Chen, senior director of Regulatory Affairs at BioDuro told PharmTech that, “FDA is advancing a shift of FIH from a discrete submission milestone to an ongoing development process jointly governed by regulators, research institutions, sponsors and clinical sites. Should the QRI (Qualified Research Institution) model achieve formal certification, it may reshape the global early-stage drug development service ecosystem.”

The pilot puts CMC review first, because pre-IND feedback spans chemistry, manufacturing, and controls alongside pharmacology/toxicology and clinical input. It ties the pilot to HHS’s Operation TrialBlazer, a response to concerns about US competitiveness against China in early-stage development. Center for Drug Evaluation and Research (CDER) Director Michael Davis, MD, PhD, noted that public comment shaped the final design. Brian Scanlan, managing partner at Freedom Bioscience Partners, said regulatory agility must be matched by faster contract development and manufacturing organization process development. Richard Graham, chairman and co-founder of TruTechnologies, urged extending risk-based, proportionate standards beyond trial start-up.²

The FDA is pairing speed with a firmer line on data integrity. In an FDA Voices commentary published September 3,³ the leaders of CDER, the Center for Biologics Evaluation and Research (CBER), the Center for Devices and Radiological Health, and the Oncology Center of Excellence stressed that good clinical practice is mandatory, particularly as reliance on foreign clinical data grows. Planned steps include more foreign bioresearch monitoring resources for early-phase trials and updated risk-based criteria by country. The FDA will also tell sponsors when a trial site can’t be inspected, give reviewers more training on informed-consent documentation, and publish inspection findings when human-subject protection or data-integrity concerns arise. It will look hardest at foreign studies not run under an IND or IDE that support US marketing applications.

Separately, an FDA analysis of first-cycle complete response rates for CDER new drug applications (NDAs) and biologics license applications (BLAs), covering FY2008–2024, found a long-term decline rather than a rise.⁴ Rates fell from roughly 47% for the FY2008 cohort to about 27% in FY2024. Approximately 77% of applications were ultimately approved across all review cycles. The analysis notes that newer BLAs have shown recent increases that warrant monitoring.

Modernizing Nonclinical Testing

On September 21, the FDA issued a direct final rule that replaces “animal tests” with “nonclinical tests” in its drug and biologics regulations.⁵ The change makes clear that non-animal methods such as human cell cultures, organs-on-chips, and computer models can support pre-human safety assessment. The rule doesn’t remove animal studies or change safety standards. The FDA also launched a database of 25 examples of new approach methodologies (NAMs) drawn from its review materials, and the comment period remains open. There are 4 validation considerations for NAMs: context of use, human biological relevance, technical characterization, and fit-for-purpose assessment.⁶

Safety, Supply, and Guidance

On September 1, the FDA required a boxed warning on Injectafer (ferric carboxymaltose injection) for symptomatic hypophosphatemia. Reports of serious harm, including fractures and hospitalization, have continued despite earlier label updates in 2020, 2023, and 2025. Fewer than 20% of patients receive phosphate testing during treatment. The FDA advises clinicians to check phosphate levels before repeat treatments within 3 months.⁷

On supply, the FDA reported that estradiol transdermal patches remain available, though specific products may vary by pharmacy.⁸ The agency is working with all 6 manufacturers, and supply has nearly doubled during the past year. Manufacturers are adding capacity and shifts, increasing batch sizes, and prioritizing patch production. Demand rose after the FDA’s November 2025 removal of boxed warnings from menopausal hormone therapy labeling.

The FDA also issued temporary compounding policies for starter parenteral nutrition products for neonates, in response to a major manufacturer’s anticipated market exit.⁹ The final guidance is effective immediately and covers outsourcing facilities and state-licensed pharmacies. Comments are accepted at any time under docket FDA-2026-D-9571.

In guidance, CDER released a draft on pharmacokinetics in patients with impaired hepatic function.¹⁰ It covers study design, data analysis, and the impact on dosing and labeling. Comments are due December 1 under docket FDA-2026-D-8693.

European Reform and Strategy

In a July 30 speech at Duke-NUS Medical School, published September 4,¹¹ European Medicines Agency (EMA) Executive Director Emer Cooke called the proposed legislative revision in the European Union “the most significant reform of medicines regulation in a generation.” She positioned regulators as enablers of innovation rather than gatekeepers. She pointed to regulatory sandboxes, biotech hub engagement, clinical trial acceleration through ACT EU and FAST-EU, and clearer AI governance.

EMA’s reform page confirms that political agreement on the overhaul was reached December 11, 2025.¹² The adopted acts enter into force in 2026, followed by a transition period through 2028, when the legislation becomes fully applicable. The reform covers innovation support, restructured scientific committees, pediatric and rare disease frameworks, shortages, and environmental sustainability. EMA plans guidance for applicants and a governance structure with 6 delivery streams.

EMA Committee Outcomes

At its September 14–17 meeting, the EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended 12 new medicines.¹³ These included Frehemgo (denecimig) for haemophilia A, Povofortay (povorcitinib) for hidradenitis suppurativa, VaxRabeo for rabies, and Zeydovio (glepaglutide) for short bowel syndrome. It also backed 2 biosimilars, 2 generics, and 11 extensions of indication, including Keytruda, Ocrevus, and Cosentyx. The committee issued no negative opinions on new medicines, but confirmed on re-examination its refusal of Xervyteg, an allogeneic fecal microbiota product.

The CHMP concluded its review of ipidacrine-containing medicines, finding that benefits still outweigh risks but recommending significant restrictions. Its concerns included poorly defined uses, limited supporting evidence, and a higher risk of adverse effects and potential liver toxicity at higher doses. It recommended removing authorization for several demyelinating and neuritic conditions and limiting prescribing to experienced specialists. It also advised caution above 80 mg daily and in patients with liver dysfunction.¹⁴

The CHMP likewise found that benefits of sodium oxybate syrup and oral solution continue to outweigh risks in alcohol dependency.¹⁵ That opinion applies to products authorized in Austria and Italy, sold under the name Alcover. Companies must run new post-authorization efficacy studies, and completing them becomes a condition of keeping the marketing authorizations. Both opinions now go to the European Commission for a legally binding decision.

On safety, the EMA’s Pharmacovigilance Risk Assessment Committee’s Aug. 31–Sept. 3 meeting started a review of injectable iron-containing medicines.¹⁶ The concern is hypophosphataemia and related bone problems, including serious cases in patients who received minimal doses and had no recognized risk factors. Overlapping symptoms with iron deficiency can delay diagnosis, and bone damage may not show on X-ray. Oral iron products are excluded. The review runs alongside FDA’s Injectafer action.

Supply and Manufacturing Implications

EMA reported a new EU supply chain for Visudyne (verteporfin).¹⁷ It adds approved manufacturing sites beyond the single US active-substance site the product had relied on. Supply is expected to be fully restored across the EU in 2027, ending the controlled distribution system. Monica Dias, EMA’s head of supply and availability of medicines and devices, said the case shows how coordination among the Commission, Member States, and the shortage working groups can ensure fair allocation and help prevent future shortages.¹⁷

PharmTech covered the EU clearance of AstraZeneca’s Trixeo Aerosphere as the first single-inhaler triple therapy for asthma in patients 12 and older.¹⁸ The are manufacturing implications, such as particle engineering, dose uniformity across 3 active ingredients, and pressure on fill-finish capacity, valves and actuators, and analytical testing. The approval coincides with the industry move toward near-zero-GWP propellants.

References

1. FDA Launches Expedited IND Pilot, Begins Accepting Applications. Press announcement. FDA. September 15, 2026. https://www.fda.gov/news-events/press-announcements/fda-launches-expedited-ind-pilot-begins-accepting-applications

2. FDA’s IND Pilot Puts CMC Review First. PharmTech. September16, 2026. https://www.pharmtech.com/view/fda-s-ind-pilot-puts-cmc-review-first

3. Good Clinical Practices Are Not Optional: FDA’s Commitment to Human Subject Protections and the Gold Standard. FDA Voices. FDA. September 3, 2026. https://www.fda.gov/news-events/fda-voices/good-clinical-practices-are-not-optional-fdas-commitment-human-subject-protections-and-gold-standard

4. First-Cycle Complete Response Rates for CDER NDA and BLA Applications: A Longitudinal Analysis, FY 2008–2024. Analysis report. FDA. Accessed September 29, 2026. https://www.fda.gov/media/194858/download?attachment

5. FDA Updates Regulations to Advance Innovative Alternatives to Animal Testing. Press announcement. FDA. September 21, 2026. https://www.fda.gov/news-events/press-announcements/fda-updates-regulations-advance-innovative-alternatives-animal-testing

6. The FDA Finalizes Rule Recognizing Non-Animal Testing Methods, Building on Year-Long Reduction Push. PharmTech. September 22, 2026. https://www.pharmtech.com/view/the-fda-finalizes-rule-recognizing-non-animal-testing-methods-building-on-year-long-reduction-push

7. FDA Adds Boxed Warning to Labeling of Ferric Carboxymaltose Injection (Injectafer) to Describe Risk of Low Phosphate Levels. Drug Safety Communication. FDA. September 1, 2026. https://www.fda.gov/drugs/drug-safety-communications/fda-adds-boxed-warning-labeling-ferric-carboxymaltose-injection-injectafer-describe-risk-low

8. FDA Update on Estradiol Transdermal Patch Availability. Drug alert/statement. FDA. September 3, 2026. https://www.fda.gov/drugs/drug-alerts-and-statements/fda-update-estradiol-transdermal-patch-availability

9. Temporary Policies for Compounding Certain Starter Parenteral Nutrition Drug Products for Neonates. Guidance. FDA. September 2026. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/temporary-policies-compounding-certain-starter-parenteral-nutrition-drug-products-neonates

10. Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling. Draft guidance. FDA. September 2026. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/pharmacokinetics-patients-impaired-hepatic-function-study-design-data-analysis-and-impact-dosing-and

11. Executive Director’s Speech at Duke-NUS Medical School, Singapore. News. EMA. September 4, 2026. https://www.ema.europa.eu/en/news/executive-directors-speech-duke-nus-medical-school-singapore

12. Reform of EU Pharmaceutical Legislation. EMA. Accessed September 2026. https://www.ema.europa.eu/en/about-us/what-we-do/reform-eu-pharmaceutical-legislation

13. Meeting Highlights from the CHMP, 14–17 September 2026. EMA. September 18, 2026. https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-14-17-september-2026

14. EMA Concludes Review of Ipidacrine-Containing Medicines. EMA. September 18, 2026. https://www.ema.europa.eu/en/news/ema-concludes-review-ipidacrine-containing-medicines

15. Benefits of Sodium Oxybate Continue to Outweigh Risks in People with Alcohol Dependency. EMA. September 18, 2026. https://www.ema.europa.eu/en/news/benefits-sodium-oxybate-continue-outweigh-risks-people-alcohol-dependency

16. Meeting Highlights from the PRAC, 31 August–3 September 2026. EMA. September 4, 2026. https://www.ema.europa.eu/en/news/meeting-highlights-pharmacovigilance-risk-assessment-committee-prac-31-august-3-september-2026

17. New EU Supply Chain to End Visudyne Shortage. News. EMA. September 4, 2026. https://www.ema.europa.eu/en/news/new-eu-supply-chain-end-visudyne-shortage

18. EU Clears AstraZeneca’s Trixeo for Asthma, Opening New Manufacturing Demand for Single-Inhaler Triple Therapy. PharmTech. September 23, 2026. https://www.pharmtech.com/view/eu-clears-astrazeneca-s-trixeo-for-asthma-opening-new-manufacturing-demand-for-single-inhaler-triple-therapy

FDA September Drug Approvals

• FDA Approves First Drug to Treat Alexander Disease
  https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-treat-alexander-disease
• FDA grants accelerated approval to sevabertinib for locally advanced or metastatic non-squamous non-small cell lung cancer
  https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-sevabertinib-locally-advanced-or-metastatic-non-squamous-non-small
• FDA grants accelerated approval to camizestrant with a CDK4/6 inhibitor for ESR1-Mutated HR-positive, HER2-negative locally advanced or metastatic breast cancer
  https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-camizestrant-cdk46-inhibitor-esr1-mutated-hr-positive-her2-negative
• FDA Approves Additional Information in Labeling for Kybella (Deoxycholic Acid) Injection Warning of Adverse Reactions Associated with Unapproved Use
  https://www.fda.gov/drugs/drug-safety-communications/fda-approves-additional-information-labeling-kybella-deoxycholic-acid-injection-warning-adverse
• FDA Approves First Therapy to Target Muscle Loss in Spinal Muscular Atrophy
  https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-therapy-target-muscle-loss-spinal-muscular-atrophy
• FDA Approves First Gene Therapy for Pediatric Patients with Sanfilippo Syndrome Type A
  https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-pediatric-patients-sanfilippo-syndrome-type
• FDA approves imlunestrant in combination with abemaciclib for ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer
  https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-imlunestrant-combination-abemaciclib-er-positive-her2-negative-esr1-mutated-advanced-or
• FDA approves belzutifan in combination with lenvatinib for advanced renal cell carcinoma with a clear cell component
  https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-belzutifan-combination-lenvatinib-advanced-renal-cell-carcinoma-clear-cell-component
• FDA Approves Drug to Treat Idiopathic Nephrotic Syndrome in Patients 2 Years and Older
  https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-drug-treat-idiopathic-nephrotic-syndrome-patients-2-years-and-older
• FDA Approves Third Treatment for Fibrodysplasia Ossificans Progressiva
  https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-third-treatment-fibrodysplasia-ossificans-progressiva

FDA September Warning Letters

• Warning Letter to Bausch & Lomb Inc.MARCS-CMS 732398 — September 04, 2026
  https://www.fda.gov/inspections-compliance-enforcement-and-criminal-investigations/warning-letters/bausch-lomb-inc-732398-09042026
• Warning Letter to Happy Farm Botanicals, Inc.MARCS-CMS 731926 — September 01, 2026
  https://www.fda.gov/inspections-compliance-enforcement-and-criminal-investigations/warning-letters/happy-farm-botanicals-inc-731926-09012026

Upcoming FDA Events

• Navigating the FDA Bayesian Draft Guidance: Design, Analysis, and Review Considerations
  https://www.fda.gov/drugs/news-events-human-drugs/navigating-fda-bayesian-draft-guidance-design-analysis-and-review-considerations-10142026
• FDA/Center for Research on Complex Generics (CRCG) Workshop on Advancing Bioequivalence Frameworks for Inhalation Drug Products: Optimizing In Vitro, In Vivo, and In Silico Methods
  https://www.fda.gov/drugs/news-events-human-drugs/fdacenter-research-complex-generics-crcg-workshop-advancing-bioequivalence-frameworks-inhalation