Rare diseases

The market approval for Fayuvi, which was rejected in 2025 due to manufacturing problems, comes shortly after Ultragenyx got the FDA nod for its first gene therapy last month.

Big Pharma needs more—not fewer—bold bets on rare and difficult diseases after Novartis’ efforts to invest in muscular dystrophy and ALS hit major roadblocks this month.

With just over $10 million in cash available to fuel operations through the end of the year, Longeveron is undertaking a strategic review after the failure of a cardiovascular study.

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.

A growing number of biotechs are entering the market with newly launched medicines, a pattern indicative of “improving capital markets,” according to Jefferies’ Andrew Tsai.

Scholar Rock’s spinal muscular atrophy drug, now to be called Isembyld, was originally rejected in September 2025, when the FDA cited compliance problems at a third-party manufacturer.

The polycythemia vera field is heating up, with Disc Medicine and Silence Therapeutics each advancing next-generation therapies, playing catch-up to Takeda and Protagonist Therapeutics, who won the FDA’s approval last month for Mimrylo.

Novartis’ shares fell 13% this week after the report of three separate late-stage asset setbacks. The impact went beyond the Swiss drugmaker, dragging peers down across the market.

Encoded Therapeutics’ lead asset ETX101 is a gene therapy being tested for Dravet syndrome. Phase 1/2 data showed substantial reductions in seizures.

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.