Ultragenyx prices Sanfilippo gene therapy at $4M; Bristol Myers shelves Orum drug
Ultragenyx’s second gene therapy approval since August gives it another sellable voucher. Elsewhere, Roche declared success in a blood cancer trial and a cell therapy maker is cutting costs.
Ultragenyx has received approval for a gene therapy to treat Sanfilippo syndrome type A, a rare disease. The therapy is called Fayuvi. According to BioPharma Dive, this is Ultragenyx's second gene therapy approval since August.
The price of the therapy has been set at $4 million, as reported by an unnamed source, though Ultragenyx did not immediately release this information to STAT News. The approval has been welcomed by patient advocates, who see it as a significant step forward in treating the disease.
Bristol Myers Squibb has discontinued development of a certain drug, BMS-986497/ORM-6151, as reported by Endpoints News. No further details were provided on the reasons for this decision.
Brief written by urgent.news from BioPharma Dive, STAT News, Endpoints News — 3 reports on this story. Machine-written — may contain errors; check the original before relying on it.
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