PTC Therapeutics : EC Approves Evrysdi To Treat Spinal Muscular Atrophy

(RTTNews) - PTC Therapeutics Inc. (PTCT) said that the European Commission has approved Evrysdi or risdiplam for the treatment of spinal muscular atrophy in patients 2 months and older.

Evrysdi will be for spinal muscular atrophy Type 1, Type 2 or Type 3 patients with one to four SMN2 copies. The EC approval is based on two studies; 1) the efficacy results from the FIREFISH study in infants aged 2 to 7 months with symptomatic Type 1 SMA, and 2) the SUNFISH study in children and young adults with Type 2 or 3 spinal muscular atrophy.

Evrysdi is based on PTC science and is commercialized in the United States by Genentech, a member of the Roche Group. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.