Roche was granted priority review by the FDA for the NDA for risdiplam (RG7916) for the treatment of spinal muscular atrophy (SMA). The Prescription Drug User Fee Act (PDUFA) goal date for a decision by the FDA is May 24, 2020. The filing acceptance by the FDA triggers a $15 million milestone payment to PTC Therapeutics, Inc.

“The FDA’s acceptance of the NDA is an important step towards making risdiplam available to SMA patients in the U.S.,” said Stuart W. Peltz, Ph.D., chief executive officer of PTC Therapeutics. “We are proud that risdiplam, the first oral small molecule targeting splicing, was produced from our proprietary splicing platform. Risdiplam’s NDA submission includes results from a broad SMA patient population, including type 1, type 2 and type 3 SMA patients demonstrating improvements in motor functions and developmental milestones, and a compelling safety profile. We believe that an oral therapeutic that reaches all affected tissues in the body would mark a significant advancement in the treatment for SMA patients and their families.”

Priority review status is designated to drugs that, if approved, would represent significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions.
The NDA filing is based on data from the dose-finding portion of studies, and clinical and preclinical pharmacokinetic and pharmacodynamic data. The double-blind, placebo-controlled trial of risdiplam in patients with type 2 or 3 SMA aged 2-25 years met its primary endpoint of change from baseline in the Motor Function Measure 32 scale.