FDA delays oral SMA therapy risdiplam as Roche readies submissions in China, EU

Roche’s oral therapy to treat spinal muscular atrophy faces a three-month delay after FDA pushed back its PDUFA date to review additional data, even as the pharma readies regulatory submissions in other territories.

In November, FDA granted Priority Review for an NDA of risdiplam from Roche (SIX:ROG; OTCQX:RHHBY) and partner PTC Therapeutics Inc. (NASDAQ:PTCT) to treat patients with spinal muscular atrophy Type 1-3, with a PDUFA date of May 24...