The FDA's industry-friendly about-face

Driving the news: The FDA has reversed several high-profile decisions made under Makary that caused discord inside and outside the administration, particularly around cell and gene therapies targeting rare diseases.

• Just this week, the Maryland biotech Regenxbio confirmed the FDA is reconsidering its experimental gene therapy for Hunter syndrome after it was rejected earlier this year.
• The FDA has also reversed course on a melanoma therapy from Replimune, and on UniQure's gene therapy for Huntington's disease, allowing both to move forward in the review process.
• "I would say these recent events ... have really been a breath of fresh air. These are dramatic, positive developments," said Tim Hunt, CEO of the Alliance for Regenerative Medicine. "It really discards the previous regime's, I would say, unhealthy fixation on methodological purity."

The agency also took the unusual step of publicly releasing a memo confirming that former top drug regulator Tracy Beth Høeg opposed an expanded approval of Sanofi's Tzield diabetes treatment for an additional pathway that's since been greenlit.

• Mike Davis, the current acting director of FDA's Center for Drug Evaluation and Research and the author of the memo, wrote that he disagreed with Høeg's assessment.

What they're saying: "The changes appear to be in the right direction but I think it hasn't been long enough to see a trajectory and know whether we can count on consistency," said RA Capital managing partner Peter Kolchinsky.

• "We've had 1.5 years of destructive chaos and decision-making by personality ... let's see the FDA function as an institution for maybe six months straight before we reach any conclusions."

The other side: "The public can have confidence in FDA's review process, as it is governed by federal statute and grounded in rigorous scientific evaluation. These scientific standards have not changed," FDA spokesperson Benjamin Nichols said in a statement.

• "The agency stands behind the career scientists and medical officers who conduct these reviews and applies the same scientific and regulatory standards consistently across all applications."

The big picture: Health and Human Services as a whole released a blueprint earlier this week for maintaining U.S. leadership in clinical trials and drug development, acknowledging America's weaknesses in the race against China.

• "Where the U.S. once dominated the earliest stages of drug discovery and human testing, it now faces competitors that are moving faster, scaling quickly, and winning the race for the investment and intellectual capital that follow," HHS wrote.
• As part of that initiative, the FDA released updated draft guidances on later-stage clinical trial processes and a series of actions aiming to speed up and streamline earlier-phase drug development.
• "We applaud FDA's efforts ... At a time when China is able to conduct clinical trials faster and cheaper than the U.S., it is imperative we advance solutions that enable America to continue leading in biopharmaceutical innovation," PhRMA spokesperson Chanse Jones said in a statement.

Meanwhile, Congress faces the task of renewing FDA user fees that are due to expire at the end of September 2027. The package also provides a venue for regulators and the drug industry to address outstanding issues.

Yes, but: All of this is happening under a temporary commissioner and acting directors of several key FDA centers. The search for permanent leadership remains underway.

• Acting commissioner Kyle Diamantas has been widely praised for calming the waters, even by past agency critics. So, too, have Davis and acting top vaccine and biologics regulator Karim Mikhail.
• "We were not pleased with previous [Center for Biologics Evaluation and Research] leadership, I will say that," Hunt said. "But I'll say the data points so far with Kyle Diamantas and Karim Mikhail have been extremely productive."

But whoever President Trump nominates for permanent leadership positions will steer the agency's longer-term priorities and culture — including addressing significant staff turnover and declining morale that many in industry say threaten the regulatory process.

• Heidi Overton, deputy assistant to the president for domestic policy, is among the candidates being eyed for the commissioner's spot, Bloomberg first reported and Axios confirmed.

What we're watching: "Even with recent indications that the agency is turning a corner, rebuilding the agency's depleted leadership ranks and replacing experienced staff lost over the past year will likely be a much longer process," Raymond James analyst Chris Meekins wrote in an investor note.