New clinical trial to Evrysdi (risdiplam) in infants with Type 1 spinal muscular atrophy (SMA)

Roche presents its new data on the SUNFISH, JEWELFISH, RAINBOWFISH study

  • Exploratory efficacy data showed 88% of infants treated with Evrysdi were alive and did not require permanent ventilation at two years
  • 59% of infants were able to sit without support for at least 5 seconds
  • No new safety signals were identified
  • In August, the FDA approved Evrysdi for the treatment of SMA in adults and children 2 months and older
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Evrysdi™ (risdiplam)

European Marketing Authorization Application for Evrysdi™ (risdiplam)

PTC announced the acceptance of the Marketing Authorization Application (MAA) by the European Medicines Agency (EMA) for Evrysdi™ (risdiplam) for the treatment of spinal muscular atrophy (SMA). The EMA previously granted PRIME (PRIority Medicines) designation to risdiplam for the treatment of people with SMA, providing a pathway for accelerated evaluation by the agency. The milestone triggers a $15 million payment to PTC from Roche.

The U.S. Food and Drug Administration recently approved Evrysdi™ (risdiplam) for the treatment of SMA for adults and children 2 months and older. Evrysdi™ (risdiplam) is based on PTC science and is commercialized in the United States by Genentech, a member of the Roche Group. Evrysdi™ is a product of the SMA collaboration between PTC, the SMA Foundation, and Roche.

Scholar Rock Announces that SRK-015 has Received Rare Pediatric Disease Designation from U.S. FDA for the Treatment of SMA

Aug. 13, 2020Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for SRK-015 for the treatment of Spinal Muscular Atrophy (SMA), a progressive, rare genetic disease that leads to motor function impairments.

Scholar Rock is on track to report 6-month interim efficacy and safety data from the ongoing TOPAZ Phase 2 clinical trial of SRK-015 in patients with Type 2 and Type 3 SMA in the fourth quarter of 2020. Top-line data for the 12-month treatment period are expected in the first half of 2021.