Regulatory

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.

The approval opens up a “modest” market for AstraZeneca’s Etcamah, according to Leerink analysts, who nevertheless looked forward to an upcoming late-stage readout that could lead to a “significantly larger opportunity” for the drug.

Belgium and the Netherlands have built some of Europe’s strongest life sciences ecosystems, powered by leading research institutions and a growing pool of experienced biotech talent. While early-stage funding remains scarce and investment is often fragmented across regions, investors see national biotech initiatives and broader EU support as catalysts for future growth.

The FDA’s primary review team found that Replimune hadn’t sufficiently addressed deficiencies that had been pointed out in the two prior rejection letters for the melanoma therapy Tudriqev.

In this episode of Denatured, you’ll hear from Christina Takke, managing partner at V-Bio Ventures and Mariette van der Velden-Roesink, founding partner at Curie Capital. We explore why the Benelux region remains scientifically strong, but investment fragmented, and what it will take to turn it into a more unified biotech investment corridor.

Last month’s FDA advisory committee meetings for therapies developed by Capricor Therapeutics and Replimune effectively illustrate the different outcomes for patients when the agency prioritizes specialization in its reviews.

Inspection access is not “an administrative footnote,” leaders of key FDA divisions wrote in an editorial on Wednesday vowing to expand oversight of foreign clinical trial sites.

The biologics license application follows months of public speculation—and regulatory reversals—for uniQure’s AMT-130. If accepted by the FDA for priority review, approval could come in the second quarter of 2027.

CSL Vifor holds the marketing authorization for Tavneos in the U.K., where the drug is marketed as Avacopan Vifor. It continues to be sold on the U.S. market by Amgen despite concerns raised by the FDA.