Rocket Pharmaceuticals Receives the US FDA’s RMAT Designation of RP-A501 for the Treatment of Danon Disease
Shots:
- The US FDA has granted RMAT designation to RP-A501, an investigational AAV-based gene therapy for the treatment of Danon Disease
- The designation was based on positive safety & efficacy data from the P-I trial (RP-A501) evaluating RP-A501 which showed that the therapy was well tolerated with evidence of restored expression of the deficient LAMP2 protein & durable improvement or stabilization of clinical parameters in the Danon Disease patients
- The outcomes show improvement or normalization across a no. of quantifiable parameters that cardiologists use in clinical practice to facilitate risk assessment and treatment decisions. The company also plans to initiate the P-II trial, expected in Q2’23
Ref: Businesswire | Image: Rocket Pharmaceuticals
Related News:- The US FDA Lifts the Clinical Hold of Rocket's RP-A501 Clinical Trial to Treat Danon Disease
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