Pharma Focus Asia

RP-A501 Gene Therapy Granted Priority Medicines (PRIME) Designation by Rocket Pharmaceuticals

Wednesday, May 31, 2023

Rocket Pharmaceuticals announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to RP-A501, the investigational adeno-associated virus (AAV)-based gene therapy. 

This therapy is designed specifically for addressing Danon Disease, an inherited cardiomyopathy that currently lacks curative treatment options and often leads to severe outcomes, including fatality.

The PRIME designation was granted based upon RP-A501 after the Phase 1 clinical trial of the therapy which demonstrated promising safety and efficacy results in individuals diagnosed with Danon Disease. The designation recognises the potential of RP-A501 to address the significant unmet medical needs of patients suffering from Danon Disease, emphasising its potential to offer substantial benefits to this specific population.

The Phase 1 trial results of RP-A501 have yielded one of the most comprehensive sets of investigational gene therapy data for any cardiac condition. Notably, RP-A501 exhibited a favourable safety profile throughout the trial. 

The data showcased consistent and significant improvements across various clinical and laboratory parameters that hold high relevance, including enhanced expression of LAMP-2 protein, reduced autophagic vacuoles, lowered levels of brain natriuretic peptide (BNP) and high sensitivity troponin I, as well as improvements in left ventricular mass and wall thickness.

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