Rocket Pharma Gets RMAT Designation for RP-A501 Treatment for Danon Disease

By Chris Wack

Rocket Pharmaceuticals Inc. said Tuesday that the U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy designation to RP-A501.

RP-A501 is Rocket's investigational adeno-associated virus-based gene therapy for the treatment of Danon Disease, a fatal genetic cardiac disease for which there are no disease-altering therapies available.

The company said RMAT designation was granted based on positive safety and efficacy data from a Phase 1 RP-A501 clinical trial, and will provide the benefits of added intensive FDA guidance and expedited review through the program's development.

The FDA's RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including gene therapies.

The initiation of a Phase 2 pivotal trial for RP-A501 is on track for the second quarter of 2023.

Results from the Phase 1 program showed RP-A501 was generally well tolerated with evidence of restored expression of the deficient LAMP2 protein, and durable improvement or stabilization of clinical parameters in the Danon Disease patients treated.

Write to Chris Wack at chris.wack@wsj.com

(END) Dow Jones Newswires

February 07, 2023 07:44 ET (12:44 GMT)

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