The Food and Drug Administration (FDA) has granted Priority Review to the New Drug Application (NDA) for risdiplam (Genentech) for the treatment of spinal muscular atrophy (SMA).

Risdiplam is an investigational, orally administered liquid survival motor neuron-2 (SMN2) splicing modifier designed to increase and sustain SMN protein levels throughout the central nervous system and peripheral tissues of the body. The NDA submission includes data from the FIREFISH and SUNFISH studies. Recently announced results from part 2 of the SUNFISH trial, which evaluated the safety and efficacy of risdiplam in patients with type 2 or 3 SMA aged 2 to 25 years, showed that the treatment met its primary end point of change from baseline in the total motor function measure 32 (MFM-32) score at Month 12.

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“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,” said Stuart W. Peltz, PhD, Chief Executive Officer of PTC Therapeutics. “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.”

A Prescription Drug User Fee Act (PDUFA) target date of May 24, 2020 has been set for the application. 

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