Rocket's gene therapy RP-L301 for rare blood disorder gets FDA regenerative medicine tag
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- The U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to Rocket Pharmaceuticals' (NASDAQ:RCKT) lentiviral-based gene therapy RP-L301 to treat Pyruvate Kinase Deficiency (PKD)
- PKD is a rare blood disorder characterized by severe anemia and excessive red blood cell breakdown. The condition occurs due to low levels of an enzyme called pyruvate kinase.
- The RMAT status was granted based on data from an ongoing phase 1 trial of RP-L301 a life-threatening disease for which currently there are no curative therapies, according to the company.
- "The first pediatric patient has shown promising initial results similar to the adults, and enrollment has been completed in the Phase 1 study," said Rocket's President and COO Kinnari Patel.
- RCKT +0.78% to $21.91 premarket May 23
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