Risdiplam-treated type 1 babies with SMA continue to achieve major motor milestones

  • All clinical trials involving risdiplam continue to demonstrate strong safety profile
  • Filing for broad labels in 2019 (US) and 1H’20 (EU)

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced data demonstrating the ongoing benefit of risdiplam (RG7916) for the treatment of all types of spinal muscular atrophy (SMA) at the 24th International Annual Congress of the World Muscle Society. Presentations include data from the FIREFISH, SUNFISH, and JEWELFISH clinical trials. The SMA program is a collaboration between PTC, the SMA Foundation, and Roche.

”We are excited that risdiplam has the potential to enter the market with a best-in-class profile for patients with all types of SMA,” said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. ”These data continue to demonstrate the disease-modifying properties of risdiplam across a broad range of ages and infants receiving the drug are continuing to experience improved motor outcomes. Importantly, these results help validate PTC’s splicing platform that is currently being used in other programs.”