Rocket Pharmaceuticals receives FDA RMAT designation for Danon disease gene therapy treatment

gene therapy

Rocket Pharmaceuticals, Inc says the U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to RP-A501.

RP-A501 is Rocket Pharmaceuticals’ 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. 

RMAT designation was granted based on positive safety and efficacy data from the phase 1 RP-A501 clinical trial and will provide the benefits of added intensive FDA guidance and expedited review through the program’s development.

“Today’s exciting RMAT designation demonstrates recognition from the FDA of the early meaningful benefit of RP-A501 in Danon disease and its potential to deliver lifesaving treatment for patients,” said Gaurav Shah, chief executive officer, Rocket Pharma. 

“We look forward to the FDA’s added guidance and support on the most efficient development and approval pathway for RP-A501, including on our anticipated phase 2 pivotal trial with the opportunity for accelerated approval.”

Shah added: “RP-A501 is the first cardiac gene therapy to receive RMAT designation from the FDA, and today’s news is another important step forward both for patients with Danon disease and for the gene therapy field. We remain on track to initiate our phase 2 trial in the second quarter and are thankful to the FDA for their continued collaboration.”

Expediting development

RMAT designation is intended to expedite the drug development and review processes for promising pipeline products, including gene therapies. A regenerative medicine therapy is eligible for RMAT designation if it is 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.

Rocket Pharmaceuticals also holds Orphan Drug (U.S.) and Rare Pediatric designations for the RP-A501 program.

The start of the phase 2 pivotal trial is on track for the second quarter of 2023. 

Rocket Pharmaceuticals’ RP-A501 phase 1 data

In phase 1, 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.

Data show consistent and robust improvements in multiple parameters including protein expression, reduced autophagic vacuoles, brain natriuretic peptide (BNP), troponin, left ventricular (LV) mass and thickness, and improved New York Heart Association (NYHA) class and Kansas City Cardiomyopathy Questionnaire (KCCQ/Quality of Life) measurements. 

The improvements and stabilization of BNP and NYHA class are in contrast to BNP increases and NYHA class deterioration observed in a representative sample of pediatric and adolescent natural history patients. Overall, the company said, the results demonstrate improvements and/or normalization across multiple quantifiable parameters that cardiologists use in clinical practice to enable risk assessment and treatment decisions.

About Danon disease

Danon Disease is a rare X-linked inherited disorder caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy. This results in accumulation of autophagosomes and glycogen, particularly in cardiac muscle and other tissues, which ultimately leads to heart failure, and for male patients, frequent death during adolescence or early adulthood. 

It is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S. and Europe. The only available treatment option for Danon disease is cardiac transplantation, which is associated with substantial complications and is not considered curative. There are no specific therapies available for the treatment of Danon disease.

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