The European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to Rocket Pharmaceuticals’ adeno-associated virus (AAV)-based gene therapy, RP-A501, for the treatment of Danon Disease, a rare X-linked inherited disorder.

The designation for the investigational gene therapy was granted based on positive safety and efficacy data from the Phase I study in patients with Danon Disease.

A favourable safety profile of RP-A501 was observed with improvements in multiple clinical and highly relevant laboratory parameters including LAMP-2 protein expression.

The study also observed reduced autophagic vacuoles, high sensitivity troponin I, brain natriuretic peptide (BNP) and left ventricular mass and wall thickness.

In addition, improvement was observed in symptoms and quality of life as measured by New York Heart Association and the Kansas City Cardiomyopathy Questionnaire, respectively.

Improvements and normalisation across several quantifiable parameters used by cardiologists in clinical practice to enable risk assessment and treatment decisions were also demonstrated in the study.

Rocket Pharma president and chief operating officer Kinnari Patel said: “PRIME designation from the EMA further highlights the positive benefit/risk profile of RP-A501 in addressing the critical unmet need of patients facing Danon Disease.

“We are thrilled by the opportunity that PRIME grants us, so that we may collaborate with our European Regulatory partners on the development of RP-A501 in the most expedient and efficient path forward.”

Reference：
https://www.pharmaceutical-business-review.com/news/ema-prime-designation-rockets-aav/