PTC Therapeutics, Inc. achieved a $20 million milestone payment from Roche under its license and collaboration agreement. The milestone payment was triggered by the first commercial sale of Evrysdi (risdiplam) in the U.S. Evrysdi was approved by the FDA on August 7, for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older.
 
Evrysdi is a survival motor neuron 2 (SMN2)-directed RNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Evrysdi (risdiplam) is based on PTC science and is commercialized in the U.S. by Genentech, a member of the Roche Group. Roche led the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.

“We are excited that Evrysdi is able to reach U.S. SMA patients so quickly following FDA approval,” said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. “This is especially relevant during the COVID-19 global pandemic, given that Evrysdi can be delivered directly to patients at home.”