This Week: Reports of tension between RFK and Makary, concerns about CNPV, 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, more reporting delves into a potential rift between the heads of HHS and FDA, ethical questions are being raised about the commissioner’s new drug voucher program, and GOP attorneys are suing FDA for approving a generic abortion drug.

Politico reported that industry and investor concerns about FDA Commissioner Marty Makary continue to cloud his tenure, following earlier reporting by The Wall Street Journal last weekend that claimed Department of Health and Human Services (HHS) Secretary Robert Kennedy Jr. and the White House discussed reducing his role. Politico also reported that Kennedy even considered replacing Makary after the recent resignation of former Center for Drug Evaluation and Research Director George Tidmarsh.

Additionally, Politico reported that the White House requested the resignation of Sanjula Jain-Nagpal, a top aide to Makary whom he had intended to promote to deputy chief of staff. Makary, however, reportedly pushed back, and although the FDA stated that she is still employed at the agency, her name was no longer listed as a health informatics specialist on its website.

STAT News published an op-ed raising concerns that the Commissioner's National Voucher Program (CNVP), promoted by Makary, may be used to prioritize drugmakers that curry favor with the administration. It also notes reporting that Tidmarsh resigned after raising concerns about the program.

STAT News also reported that, according to its sources, when FDA voted whether to approve the first product for the CNVP, the voting members did not include the review team. The decision was instead made by agency leaders, raising concerns that it was more of a political move than a scientific one.

Reuters reported that three Republican-led states, Missouri, Kansas, and Idaho, broadened their lawsuit against the federal government to block FDA's recent approval of Evita Solutions' generic version of the abortion drug mifepristone. The lawsuit broadly argues that FDA acted improperly in easing restrictions on Mifepristone by allowing it to be prescribed remotely and received through the mail.

A group of academic and legal scholars published an article in the Journal of the American Medical Association (JAMA) Internal Medicine examining the future of generative artificial intelligence (gen-AI). They argue that current regulatory frameworks are insufficient to provide adequate oversight and that a licensure framework may help oversight keep pace with the evolving technology.

BioSpace reported that Jeffries, the financial services company, found that more than 94% of drugs that receive breakthrough designation go on to earn priority review. Its analysis follows an October report that also found that the agency eventually approves 72% of drugs with breakthrough designation.

Fierce Biotech published an article detailing FDA and HHS has engaged with stakeholders in the cell and gene therapy (CGT) space to ease regulatory burdens and accelerate CGT products coming to market. It comes after Makary and Vinay Prasad, director of the Center for Biologics Evaluation and Research (CBER), published an article in the New England Journal of Medicine (NEJM) proposing a new pathway for CGTs.

The next public meeting to discuss reauthorizing the Biosimilar User Fee Act (BsUFA) for fiscal years 2028 through 2032 is scheduled for 3 December. FDA is hosting the hybrid public meeting to kick off the next round of BsUFA negotiations.

FDA announced it has published additional draft and revised draft product-specific guidances that address topics such as the design of bioequivalence (BE) studies to support abbreviated new drug applications (ANDAs). It published 30 new drug-specific draft guidances and 40 drug-specific revised guidances.

Drugs & Biologics

FDA announced its Office of New Drugs has been piloting a program since October to streamline sponsor communications after holding formal meetings that will allow them to get a “Meeting Minute Clarification Opportunity.” Pilot participants can email agency staff with their questions, aiming to receive a response within three business days.

FDA issued a safety communication stating that it has received a report of a pediatric patient with congenital thrombotic thrombocytopenic purpura treated with Takeda Pharmaceuticals' Adzynma (ADAMTS13, recombinant-krhn) who has died. The agency said before getting the treatment, the patient had severe allergic reactions to fresh frozen plasma (FFP), and it is investigating the risk of developing neutralizing antibodies from the drug that could be fatal.

Amgen's cancer drug Imdelltra (tarlatamab-dlle) received full approval from FDA to treat adults with extensive stage small cell lung cancer (ES-SCLC) with disease progression on or after platinum-based chemotherapy. The drug had previously received accelerated approval for the indication in 2024.

AstraZeneca's cancer drug Koselugo (selumetinib) also received approval to treat adults with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). The drug had previously been approved to treat pediatric patients one years old and older.

Bayer's cancer drug Hyrnuo (sevabertinib), a kinase inhibitor, received accelerated approval from FDA to treat locally advanced or metastatic, non-squamous non-small cell lung cancer (NSCLC) whose tumors have HER2 (ERBB2) tyrosine kinase domain (TKD) activating mutations and who have received a prior systemic therapy. The agency also approved Life Technologies' Oncomine Dx Target test as a companion diagnostic to the drug.

Janssen Biotech's Darzalex Faspro (daratumumab and hyaluronidase-fihj) in combination with bortezomib, cyclophosphamide, and dexamethasone (VCd) has received full approval for the treatment of newly diagnosed light chain (AL) amyloidosis. The combination drug had initially received accelerated approval for the indication in 2021.

The Duke-Margolis Institute for Health Policy and FDA will co-host a hybrid public workshop entitled “Improving Anaphylaxis Outcomes: Approaches for Enhancing Access to Epinephrine" on 16 December. The workshop aims to foster public discussion on expanding access to and use of epinephrine.

Indian drugmaker, Cdymax Pharma, received a warning letter from FDA for failing to meet current good manufacturing practice (CGMP) requirements. More specifically, the company was cited for failing to investigate and document out-of-specification results adequately and for not implementing appropriate corrective actions. It also reportedly failed to ensure that its active pharmaceutical ingredients (APIs) conformed to quality and purity standards.

The Office of Prescription Drug Promotion (OPDP) issued an untitled letter to SK Life Science for a direct-to-consumer television advertisement for its seizure drug Xcopri (cenobamate) for failing to include warnings and precautionary information about potential liver injury. While the ad included some risks, the agency stated that it failed to disclose risks such as side effects on the electrical system of the heart.

OPDP also issued an untitled letter to PharmaTher for making false or misleading statements on its website when marketing ketamine hydrochloride injections. The agency said the company failed to communicate the risks of the product and the full indication approved by FDA.

Medtech

FDA announced that the General Hospital and Personal Use Devices Panel meeting, originally scheduled for October 8, will now be held virtually on December 10. The panel of external experts will discuss the use of germicidal ultraviolet (UV) light as a mode of disinfection.

Wired reported that Neuralink competitor Paradromics has received FDA approval to begin testing its brain implants in an early-stage human trial. The implants are designed to help individuals with severe motor impairments regain their ability to communicate.

This Week: Reports of tension between RFK and Makary, concerns about CNPV, and more

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