Sarepta Therapeutics Inc. (NASDAQ:SRPT) has announced the completion of a Type C ‘written response only’ meeting with the FDA’s Centre for Biologics Evaluation and Research’s (CBER) Office of Tissues and Advanced Therapies(OTAT) for SRP-9001. The company seeks to get concurrence from OTAT to commence the next SRP-9001 clinical trial using commercial process material.
Sarepta to provide OTAT with necessary assays and data on SRP-9001
SRP-9001 is the company’s investigational gene therapy for Duchene muscular dystrophy treatment. OTA has requested the utilization of more potency assay for SRP-9001 commercial process material release before dosing in the clinical study. Currently, Sarepta has numerous assays and data which it will employ in response to the request from OTAT. However, there is a need for more engagement with the agency to ascertain the potency assay approach’s acceptability.
Doug Ingram, the Chief Executive Officer and President of Sarepta stated that they are looking forward to collaborating with OTAT to meet the requests and clarify the timing for the commercial supply study’s start. Ingram added that they plan to offer more updates on the same as they can. He further explained that each day, thousands of kids worsen due to the irreversible damage that Duchenne muscular dystrophy causes. Therefore it is for this reason that Sarepta is committed to working with the division to meet the OTAT request. This continues advancing the potentially transformative gene therapy for this patient population.
Sarepta has exclusive rights to the micro-dystrophin gene therapy program
Sarepta’s investigational gene therapy has been designed to deliver a micro-dystrophin-encoding gene to the muscle tissue targeting micro-dystrophin protein production. The company is responsible for the development and global manufacturing if SRP-9001, and it has plans for commercializing the therapy in the US.
In December last year, Sarepta announced that it had granted Roche exclusive rights to commercialize and launch SRP-9001 outside the US. The company has exclusive rights to the gene therapy program, which was initially developed at Nationwide Children’s Hospital at Abigail Wexner Research Institute.