This Week at FDA: ‘Simple reform’ reorg, adcomm updates, and more

Welcome to another installment of This Week at FDA, your weekly source for updates – big and small – on FDA, drug, and medical device regulation, and what we’re reading from around the web. This week, FDA announced a major reorganization intended to streamline operations, and the agency’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTAGTAC) met this week to vote on whether to recommend therapies for Duchenne muscular dystrophy and melanoma.

Earlier this week, FDA announced it will reorganize most of its product centers and centralize certain agency functions following staffing cuts by the Department of Government Efficiency (DOGE) last year. NOTUS reported that the organizational and functional changes will take effect on 1 October, including stripping the agency’s product centers of their individual human resources, finance, and information technology services in favor of centralized offices for those functions. NOTUS also reported that the agency will stop assigning specialized field inspectors to specific subject areas and instead will have them operate as generalists conducting inspections and investigations across multiple industries.

The Cellular, Tissue, and Gene Therapies Advisory Committee (CTAGTAC) met this week and voted 9-3 against recommending Capricor Therapeutics’ Duchenne muscular dystrophy cell therapy, deramiocel, in what BioSpace described as a chaotic and contentious meeting. In FDA briefing documents, the agency said the drug did not meet the prespecified primary and secondary efficacy endpoints, which was contested by the company.

The following day, CTAGTAC voted 10-3 in favor of Replimune's RP1 advanced melanoma drug, STAT reported. The drug was previously rejected twice by FDA, and the agency is expected to make an approval decision by 2 August.

President Donald Trump issued an executive order barring executive departments and agencies from funding gain-of-function research on biological agents and pathogens, as well as other high-risk life sciences research conducted by foreign entities in countries of concern, arguing that the method could endanger lives. The announcement comes the same week as Anthony Fauci, former director of the National Institute of Allergy and Infectious Diseases (NIAID), testified before Congress about the COVID-19 pandemic, which some Republicans believe was caused by a US-funded gain-of-function study that leaked from a lab in Wuhan, China, despite a lack of any evidence.

The Wall Street Journal reported that Trump has been pushing Health and Human Services Secretary Robert Kennedy Jr. to do more to investigate the alleged connection between vaccines and autism, according to anonymous sources. One of the top priorities the president conveyed to the secretary was to reduce the number of childhood vaccine shots that are recommended per federal guidelines.

FDA is reopening the comment period for a rule the agency proposed in May to remove the term “gender” and replace it with “sex” or delete the reference altogether to comply with an executive order from January 2025 on gender ideology. The original comment period closed on 6 July but will now be open until 2 October.

FDA published several guidances this week, including finalizing a trio of guidances to promote greater participation and diversity in oncology clinical trials. The agency also published a draft guidance for sponsors developing container closure systems and device constituent parts for proposed biosimilar and interchangeable biosimilar products. Furthermore, it published two guidances on transdermal or topical delivery systems (TDS), including a final guidance on assessing adhesives for TDS products and a draft guidance on assessing skin irritation using in vivo studies in TDS products.

The Office of Management and Budget received a final rule on nonclinical testing terminology as part of the agency's efforts to reduce reliance on animal testing. When approved, the rule would add nonclinical testing to the definition sections of FDA's drug and biological product regulations, substitute nonclinical for animal in phrases such as animal test, and make other changes in the safety and reporting sections of its regulations.

Experts on FDA's Pediatric Advisory Committee are scheduled to convene virtually on 16 September to discuss post-marketing pediatric-focused safety reviews for more than two dozen products. Two of the products to be reviewed are under the oversight of CBER, 10 products are under CDRH, and 15 products are under CDER.

FDA published user fee rates for FY 2027 across its prescription drug, generic drug, biosimilar, medical device, and over-the-counter drug programs. It also published FY 2027 rates for other activities, including outsourcing facility fees.

Drugs & Biologics

The Department of Health and Human Services announced that it is releasing $600M in Congressionally appropriated funds for Gavi, the Vaccine Alliance (Gavi), for FY 2025 and FY 2026, after the global public-private vaccine distribution organization reportedly committed to meaningful reforms. The department said that Gavi has committed to transitioning away from mercury-containing vaccines and expanding access to mercury-free alternatives.

Vascular Solutions' biologics license application (BLA) for Ezplaz Freeze Dried Plasma (FDP) was approved by FDA as the first freeze-dried plasma product available in the US. The plasma is intended for adult patients who need plasma transfusion and who don't have other plasma products available.

The Center for Biologics Research and Evaluation (CBER) updated its Standard Operating Policy and Procedure (SOPP) for processing biologics license applications (BLA) and new drug applications (NDA). The agency updated a reference code JA 910.16 to now reference SOPP 8401.10: Processing the Summary Basis of Regulatory Action.

Medtech

FDA has classified phase-changing fiducial markers for radiation therapy as class II devices with special controls. The agency will require mitigation measures for the devices, including biocompatibility evaluation, animal performance testing, clinical and non-clinical performance testing, sterilization validation, shelf-life testing, and meeting labeling requirements.

Argon Medical issued a Class I recall for certain Argon Medical convenience kits containing Spectra Medical's pain management kits with Huons lidocaine HCl injection due to concerns about contamination that may lead to infections, inflammation, and reduced anesthetic effect. As of June 1, FDA said the company reported two serious injuries related to the recall but no deaths.