Government

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.

Though the potential change is unconfirmed, media outlets reported Wednesday that one deputy commissioner would focus on the intersection between health and AI, while another will be dedicated to drugs.

The current classifications of vaccine recommendations could imply that not all shots require the consent of patients or their parents, according to Health Secretary Robert F. Kennedy Jr.

In this episode of Denatured, you’ll hear from Ian McGowan, Chief Medical Officer at Synklino and James Hunter, associate professor of transplantation at Oxford University. We explore transplantation and antiviral innovation and why CMV remains one of the most persistent and consequential challenges facing transplant patients today.

If Heidi Overton clears the Senate nomination process, analysts say she will have a tough job ahead to “restore predictability” to an agency battered by layoffs and controversial policies.

Despite ranking among the world’s top scientific producers, Italy has struggled to turn research into startups. Now, stronger tech transfer offices, private capital and returning scientists are pushing the country toward a more mature innovation economy.