Ionis Pharmaceuticals secures the first targeted approval for Alexander disease, marking a significant milestone in rare disease treatment. Meanwhile, Novo Nordisk and Novartis face setbacks with late-stage trial cancellations and Phase 3 failures in cardiovascular and muscular dystrophy indications.
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.
Original title: “Novo’s canned heart disease trials, Novartis’ clinical fails, first approval for Alexander disease”