FDA makes good on pledge on drug rejection letters

The Food and Drug Administration is now publishing complete response letters to drugmakers shortly after they are issued, marking a major shift in transparency into regulation by the agency. Yesterday, the FDA disclosed 89 previously unreleased CRLs from 2024 and 2025, including rejections for Replimune, Capricor Therapeutics, and Ultragenyx.

Traditionally, it’s been up to companies to disclose the reasons for an FDA rejection, a process that critics contend can lead drugmakers to misrepresent what the agency said. In July, the agency announced its intention to release letters in “real time,” and published a batch of letters for drugs that had eventually won approval. Yesterday’s release went beyond that.

Notably, one of the CRLs was for Lykos Therapeutics, the company attempting to win approval for MDMA-assisted psychotherapy for PTSD. While most of the letter’s complaints were addressed during the contentious advisory committee last summer, the FDA recommended using an "evidence-based standard of care psychotherapy in lieu of the MAPS-developed psychotherapy in any future studies."

gene therapy

Patient who helped pioneer gene therapy dies at 62

Donavon Decker, a soft-spoken but relentless advocate for muscular dystrophy patients, died this week at 62 from complications of an elevator accident compounded by his disease. In 1999, he became the first muscular dystrophy patient to receive a gene therapy, STAT’s Jason Mast writes, volunteering for a risky experiment that advanced the field without offering him any personal benefit.

That tiny foot injection helped prove genes could be safely delivered into muscle, paving the way for today’s approved therapies for spinal muscular atrophy and Duchenne. Though treatments for his condition, limb-girdle muscular dystrophy, remain elusive, Decker spent decades lobbying Congress, advising the FDA, and inspiring families to pursue independent efforts.

“Sometimes I feel overwhelmed when I see nothing happening, and I can see how some companies are not working on the research like they say they do,” Decker said once in a documnetary about his life. “That makes me angry at times and sometimes it gets the best of me, but it also makes me a strong advocate.”

psychedelics

LSD eases anxiety in mid-stage trial

LSD reduced symptoms of generalized anxiety disorder in a midstage study, marking the first modern trial of the psychedelic to show sustained benefits without psychotherapy. Nearly 200 patients received one of four LSD doses or placebo in study led by the biotech MindMed.

The 100-microgram dose led to significant improvements: 65% of patients reported lasting relief at three months and nearly half entered remission.

Side effects included hallucinations, nausea, and headaches, and most participants correctly guessed whether they got LSD, undercutting trial blinding. The results, published in JAMA, revive decades-old research and could advance LSD toward FDA approval — though larger, late-stage trials are still needed to confirm durability and safety.

podcast

New stakes in the vaccine dispute and a boost for biotech

Does President Trump deserve a Nobel Peace Prize for Operation Warp Speed? What will health secretary Robert F. Kennedy Jr. tell Congress about the changes at the Centers for Disease Control and Prevention? And is biotech back?

We discuss all that and more on the latest episode of "The Readout LOUD," STAT's weekly biotech podcast.

Labor Day is in the rearview mirror, and that means Adam, Elaine, and Allison are all back to work. They take the pulse of the biotech market and discuss forthcoming data readouts at two sleep and lung disorder conferences. But first, they discuss the bubbling uncertainty in the vaccine field.

Listen here.

HIV

PEPFAR to distribute Gilead’s HIV prevention drug

The Trump administration yesterday confirmed that it will work with Gilead Sciences and The Global Fund to Fight AIDS, Tuberculosis and Malaria to provide lenacapavir, a groundbreaking HIV prevention drug, to up to 2 million people in low- and middle-income countries, STAT's Ed Silverman and Jason Mast write.

The ambitious effort was first announced late last year, but doubts quickly emerged after the Trump administration began to drastically cut foreign aid. The move appeared to jeopardize the future of the President’s Emergency Plan for AIDS Relief, or PEPFAR, which has long been the world’s leading provider of HIV prevention drugs. Only about half of its $6 billion budget has reportedly been appropriated.

The deal is meant to be a bridge to a longer-term solution that Gilead outlined last year, when it announced an agreement to allow half a dozen generic manufacturers to produce and sell its drug royalty-free in 120 low- and middle-income countries.

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