Rocket Pharmaceuticals Gets RMAT Designation for Danon Disease Gene Therapy

February 9, 2023

The FDA has granted regenerative medicine advanced therapy (RMAT) designation to Rocket Pharmaceutical’s investigational adeno-associated virus (AAV)-based gene therapy for the treatment of Danon disease, a devastating and fatal genetic cardiac disease for which there are no disease-altering therapies available. 

Drugs are eligible for the RMAT designation if they are intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition.

RMAT designation was granted based on positive safety and efficacy data from a Phase 1 clinical trial. 

RP-A501 also holds orphan drug and rare pediatric designations. The drug is the first cardiac gene therapy to receive RMAT designation, and is on track to initiate its Phase 2 trial in the second quarter, the company says.

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