This is just an interesting read for anyone interested or those who want to get a slightly clearer view of the opportunities available if certain pathways are taken, for some companies.
I am not suggesting we are the same, or whether we have taken the same pathways, but you can see the infrastructure more clearly and how it can fit together.
And the further disclaimer is that even if you choose this course, regulatory bodies may not come to the party.
CAMBRIDGE, Mass.--(
BUSINESS WIRE)--
Scholar 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.
Scholar Rock announces that SRK-015 has received Rare Pediatric Disease Designation from U.S. FDA for the treatment of Spinal Muscular Atrophy
Tweet this“This Rare Pediatric Disease designation along with the previously granted Orphan Drug Designation highlights the FDA’s recognition of the unmet medical needs of patients with SMA. We continue to aim to establish SRK-015 as the potential first muscle-directed therapy to address motor function deficits that persist despite the availability of SMN upregulators,” said Yung Chyung, M.D., Chief Medical Officer of Scholar Rock. “We are encouraged by the progress of the TOPAZ Phase 2 trial and look forward to the interim read-out next quarter that will provide important insights into the potential of SRK-015 in patients with SMA.”
The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 individuals in the United States. If a biologics license application (BLA) for SRK-015 for the treatment of SMA is approved by the FDA, Scholar Rock may be eligible to receive a priority review voucher, which may be redeemed to obtain priority review for any subsequent marketing application or be sold or transferred.
(20min delay)