The FDA Group's Insider Newsletter

The FDA Group's Insider Newsletter

RA/QA News Roll: Late September 2025

Here's what's happening at FDA and across the broader life science RA/QA space.

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The FDA Group
Oct 06, 2025
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This is our biweekly recap of news and industry happenings curated for our paid subscribers. Our goal is to bring you the headline news and the stories, research, and insights that might be harder to find.

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Here are some key items from the past couple of weeks.

💊 = Pharma, biologic, and biotech.
💉 = Medical device, medtech, diagnostics, and digital health.

FDA and the New Administration


  • Government shutdown is here — At midnight on Oct. 1, the government officially shut down after legislators failed to pass an annual appropriations package. While the FDA will fare better than most agencies (with about 86% of staff expected to remain working, albeit unpaid), certain activities will pause. The agency cannot accept new applications from life sciences companies during the shutdown, though reviews of existing applications may continue. The FDA clarified that activities “related to imminent threats to the safety of human life or protection of property” will continue.

  • FDA exempt from additional RIFs during shutdown — Addressing staff concerns about potential layoffs, FDA Commissioner Marty Makary told employees in a video that he had secured a White House exemption from further reductions in force (RIFs). “I was able to ensure an exemption, because we are in the business of public safety,” Makary said.

  • Makary distances himself from controversial drug announcements — Makary has come under scrutiny over two recent regulatory moves:

    • The FDA announced it had initiated a label change for acetaminophen (Tylenol and similar products) to reflect evidence suggesting that use during pregnancy may be linked to higher risks of autism and ADHD in children. The agency acknowledged that a causal relationship has not been established and that there are contrary studies.

    • Makary also announced the FDA would move toward approving leucovorin, a folate derivative, for use in children with autism — a step critics argue is premature given the current evidence.

  • No new “FDA Direct” episodes released — Between May 8 and August 27, Makary appeared in 16 episodes of FDA Direct, often releasing one or two per week. Since then, no new episodes have been published. The program appears to have been deprioritized, possibly due to low viewership or Makary’s packed speaking schedule.

  • White House overrides planned autism announcements — According to CNN, President Trump bypassed his health department’s careful plans to roll out a nuanced autism initiative. Officials had anticipated a modest announcement: a warning on Tylenol, a $50 million research commitment, and approval of a potential treatment. Instead, Trump commandeered the event to claim sweeping victory, catching administration officials off guard.

FDA General


  • LDT rule officially rescinded 💉 — The FDA published a notice in the Federal Register that it is vacating its 2024 LDT (laboratory-developed tests) final rule and reverting to the prior regulatory framework. This follows a court decision earlier this year that vacated the rule as unlawful.

  • Major drug advertising crackdown continues 💊 — The FDA released multiple “Untitled Letters” to drug manufacturers alleging their direct-to-consumer ads lacked sufficient support. Some letters argued that clinical trials had not demonstrated the advertised outcomes, such as restoring pre-disease quality of life.

  • Two CGT guidances advance to White House review 💊 — The FDA sent two draft guidance documents to the White House: one on “Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations,” and another on “Postapproval Methods to Capture Safety and Efficacy Data for Cell and Gene Therapy Products.” These are part of the agency’s commitments under the current Prescription Drug User Fee Act.

  • Tidmarsh announces, then retracts, surrogate endpoint review 💊 — George Tidmarsh, Director of CDER, posted that while surrogate endpoints have accelerated treatments, they also risk failures in confirmatory trials. Hours later, he retracted that post, clarifying it reflected his personal views only and not those of the FDA or HHS.

  • FDA announces leucovorin label expansion for rare condition 💊 — The FDA revealed it will rescind the prior withdrawal of leucovorin (originally marketed by Glaxo Wellcome until 1999) and restore its label to cover treatment of autism-like symptoms in patients with cerebral folate deficiency, an ultra-rare disorder. At a White House press event, officials spoke of broader autism applications, though the agency’s statement clarifies the indication remains narrowly defined.

  • OMUFA program officially lapses 💊 — The Over-the-Counter Monograph Drug User Fee Act (OMUFA), which funds FDA review of nonprescription drugs via user fees, expired at midnight at the beginning of October. That lapse could slow reviews and funding for OTC drug oversight.

  • Notable approval for Stealth’s elamipretide 💊 — On September 19, 2025, the FDA granted accelerated approval to elamipretide (now marketed as Forzinity) for Barth syndrome, awarding a Rare Disease Priority Review Voucher. This marks the FDA’s willingness to expedite approvals in high-risk, small-population scenarios. See Stealth’s announcement and BioPharma Dive’s reporting.

  • FDA announces meetings on AI and generics 💊 — The FDA, together with the Center for Research on Complex Generics, scheduled a meeting on October 15–16 focused on “opportunities, applications, and regulatory considerations of Artificial Intelligence (AI) in generic drug development and lifecycle management.” They also published a slate of 2026 meetings, including one addressing how to navigate the generic GLP-1 pathway.

  • PreCheck program faces pressure test 💊 — President Trump declared that pharmaceutical companies will face 100% tariffs on branded or patented products unless they are building U.S.-based manufacturing facilities. This puts significant pressure on the FDA’s recently adopted PreCheck Program, which is intended to expedite domestic facility approvals, to deliver results.

  • Digital Health Advisory Committee to discuss AI in mental health 💉 — The FDA’s Digital Health Advisory Committee is set to convene in November to advise on potential regulatory frameworks for generative AI–enabled mental health devices, building on discussions from its inaugural meeting last year.

Compliance and Enforcement


  • Warning letter to Hims & Hers cites implied claims 💊 — On September 9, the FDA sent Hims & Hers a Warning Letter calling out advertisements for compounded semaglutide that “imply that your products are the same as an FDA-approved product when they are not,” since compounded drug products are not FDA-approved.

  • New warning letter following stopper complaints 💊 — The FDA released a Warning Letter to Janssen Vaccines Corp. following an inspection of its Incheon, South Korea facility, citing inadequate investigations of stopper-related complaints and lapses in regulatory notification of product quality defects.

  • Actor portrayals flagged in advertising letters 💊 — Many of the FDA’s recent Untitled Letters criticize advertisements for casting actors as patients in such a way that they appear symptom-free during treatment, suggesting overpromised efficacy. For instance, Teva was cited for an Austedo XR ad in which actors “go through their daily activities without showing involuntary movements that are easily noticed by the viewer.”

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