The Regenerative Medicine Advanced Therapies designation of RP-A501, which happens to be an adeno-associated virus-based gene therapy when it comes to the treatment of Danon disease, has been granted by the US Food and Drug Administration.
According to Dr. Gaurav Shah, one of the chief executives of a pharma firm, RP-501 is the first gene therapy related to the heart that has received the designation of RMAT from the FDA. Due to this designation, there shall come added benefits of an intensive FDA guidance and also a quick review of the program’s development.
The RMAT designation has come into play in order to quicken drug development and also review the process when it comes to pipeline products that are promising, such as gene therapies. It is well to be noted that a regenerative gene therapy is a fit for the RMAT designation if it is focused on treating, modifying, reversing, or curing a serious or life-threatening condition, and the preliminary data suggests that the therapy or the drug, both have the potential to take care of the unmet medical needs of the condition and the disease.
Danon disease happens to be a genetic, rare disease that is caused by a mutation in the gene encoding lysosome-associated membrane protein 2, or LAMP-2, which is a crucial autophagy mediator. This, ultimately, amounts to heart failure and is mostly fatal to the male patients. Besides the cardiac transplant, which is not regarded as curative, there are as of now no disease-altering treatments that can be looked into.
The Phase I trial
This RMAT designation has been granted based on the favourable efficacy and safety data that one could get from the Phase I RP-A501 clinical trial. The inferences from the phase I trial give away one of the most absolute investigational gene therapy datasets in the case of any cardiac condition.
The toleration level of RP-A501 was high in the trial. The evidence also showed a restored deficient LAMP2 protein expression and also a durable enhancement of stabilisation in terms of clinical parameters.
It is also crucial to note here that stabilisation as well as improvements in the brain natriuretic peptide and New York Heart Association class are in complete contrast to the rise in BNP and NYHA class deterioration that has been observed in the sample given in the cases of adolescent and paediatric natural histories of patients.
A Phase II trial is also in the works for this year’s second quarter. This is planned as a single-arm, open-label trial with a composite endpoint based on a biomarker and also a natural history comparator.