May 19, 2024

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FDA Advisory Committee Meeting on the first DMD gene therapy SRP-9001 is set for May 12th

FDA Advisory Committee Meeting on the first DMD gene therapy SRP-9001 is set for May 12th



 

FDA Advisory Committee Meeting on the first DMD gene therapy SRP-9001 is set for May 12th. 

On April 10th, Sarepta Therapeutics announced that the US Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee has set a date of May 12, 2023, for the meeting to review the Biologics License Application (BLA) of SRP-9001 (delenexin gene moxeparvovec). The Advisory Committee meeting will be held in a virtual format.

 

FDA Advisory Committee Meeting on the first DMD gene therapy SRP-9001 is set for May 12th. 

 

 

SRP-9001 is a gene therapy under development by Sarepta for the treatment of Duchenne muscular dystrophy (DMD). If approved, it would be the world’s first gene therapy product for DMD.

 

“We look forward to sharing compelling evidence with the Advisory Committee on May 12, 2023, supporting the transformative potential of SRP-9001 for the treatment of DMD,” said Doug Ingram, Sarepta’s President, and CEO. “We again thank the Center for Biologics Evaluation and Research and the Office of Tissues and Advanced Therapies for their swift work in scheduling the Advisory Committee prior to our regulatory action date of May 29, 2023.”

 

Designed to deliver SRP-9001 to muscle tissue, the therapy targets functional components that produce the dystrophin protein.

Sarepta is responsible for global development and manufacturing of SRP-901 and plans to commercialize SRP-9001 in the US following FDA approval.

In December 2019, Roche partnered with Sarepta to combine Roche’s global reach, commercial presence, and regulatory expertise with Sarepta’s Duchenne gene therapy candidate to accelerate access to SRP-9001 for patients outside the US.

 

SRP-9001 is an investigational gene therapy being developed by Sarepta for the treatment of Duchenne muscular dystrophy (DMD). If approved, it will be the world’s first DMD gene therapy product.

 

Doug Ingram, President and CEO of Sarepta, said: “We look forward to sharing the wealth of evidence supporting the transformative potential of SRP-9001 for the treatment of DMD with the Advisory Committee on May 12, 2023. We thank the Center for Biologics Evaluation and Research and Therapeutics once again. The Product Office worked expeditiously to schedule the Advisory Committee ahead of our regulatory action date of May 29, 2023.”

 

Designed to deliver SRP-9001 into muscle tissue, targeting the functional components that produce dystrophin.

Sarepta is responsible for the global development and manufacturing of SRP-901 and plans to commercialize SRP-9001 in the United States upon FDA approval.

 

In December 2019, Roche partnered with Sarepta to combine Roche’s global presence, commercial presence and regulatory expertise with Sarepta Duchenne’s candidate gene therapy to accelerate access to SRP-9001 for patients outside the United States.

 

 

The first DMD gene therapy SRP-9001 may cost 4 million US dollars

DMD gene therapy phase 2 clinical trials are not effective

 

 

 

FDA Advisory Committee Meeting on the first DMD gene therapy SRP-9001 is set for May 12th

(source:internet, reference only)


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