Rocket Pharma Gets RMAT Designation for RP-A501 Treatment for Danon Disease
February 07 2023 - 07:59AM
Dow Jones News
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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