Government

In Thursday’s Senate committee meeting, FDA commissioner nominee Heidi Overton was repeatedly questioned about her ability to retain the FDA’s independence under President Donald Trump, as well as her views on vaccines and abortion drugs.

Novo plans to hit $23B in revenue by 2035, has further reduced its workforce by 4,000 and is boosting manufacturing capacity for its Wegovy pill, but investors are unconvinced it will be enough to turn things around; Viking Therapeutics and Roche report positive results for their weight loss assets; Ultragenyx snags FDA approval for its Sanfilippo syndrome gene therapy; IPOs keep on coming.

While increases in the interest rate from the Federal Reserve are usually bad for biotech, the sector at this moment is poised to withstand the blow, according to analysts, though earlier stage companies may be left in the lurch.

The FDA held a public forum on Monday to discuss the future of psychedelic therapeutics, followed by a meeting on Wednesday regarding recommendations for reauthorizing the Prescription Drug User Fee Act the day after launching its TrialBlazer initiative. Meanwhile, Definium Therapeutics notched another late-stage trial win for its LSD drug, and Novartis continues to reel from the company’s triple asset stumble.

Newly solidified in their roles as the heads of CBER and CDER, Karim Mikhail and Michael Davis have been working for months to right the ship that had begun to capsize under former Commissioner Marty Makary. Meanwhile, Jared Seehafer will become the first-ever deputy commissioner focused on artificial intelligence.

September has been a rough month already for Novo Nordisk, which canned two more late-stage trials of its embattled IL-6 inhibitor, and Novartis, which took Phase 3 hits in both cardiovascular and muscular dystrophy indications; Ultragenyx’s big bet in Angelman syndrome failed to yield benefit in Phase 3 but Ionis’ Pharmaceuticals nabbed the first targeted approval for Alexander disease; and on the business side, biopharma investment is back in a big way.

The decision to promote Michael Davis and Karim Mikhail from acting to permanent division heads comes as the FDA awaits the potential confirmation of Heidi Overton, President Donald Trump’s nominee for commissioner.

A new program from ARPA-H has devoted funding to make individualized RNA-based genetic medicine more efficient to produce and improve accessibility.

Novartis hits pause on several CAR T trials after three patient deaths, followed by Bristol Myers Squibb after separate safety signals; Revolution Medicines scores early FDA approval for groundbreaking pancreatic cancer drug; BioNTech suspends a mid-stage trial of its mRNA cancer immunotherapy after a futility analysis; and nine mid-sized biopharma companies join their big pharma peers in President Donald Trump’s Most Favored Nation drug pricing scheme.

Astellas Pharma, BridgeBio, BeOne Medicines and six other large biotechs join the 17 Big Pharmas that previously signed on to President Donald Trump’s Most Favored Nation (MFN) drug pricing program, representing some 89% of the branded drug market.