Another failed study in Duchenne muscular dystrophy spells the end for edasalonexent and possibly the firm.

Phase II results look good for one of the first-in-class product’s potential indications, C3 glomerulopathy. The product was highlighted late last year as an up-and-coming pipeline asset.

Positive Phase II data for Aptinyx’s novel NMDA receptor modulator are strong enough to take the product into a pivotal study in this difficult-to-treat area.

Orchard Therapeutics is on the cusp of the first approval for its ex vivo gene therapy, Libmeldy, for metachromatic leukodystrophy.

Gilead/Kite’s second CAR-T product is given the go-ahead for approval by the CHMP for mantle cell lymphoma.

A Phase III trial of Swedish Orphan Biovitrum’s Doptelet in solid tumor cancer patients with chemotherapy-induced thrombocytopenia has failed. The setback could affect the firm’s growth expectations.