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.

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Mia

Light a candle, let it burn. 🕯 Never give up the hope, let the darkness disapear. 🌅 Had a son with X-Linked Spinal Muscular Atrophy (SMAX2)