Experimental Gene Therapy on the Fast-Track to U.S. Approval

After one family’s story behind an experimental gene therapy was a success, U.S. biotech Sarepta is on its way to approval by U.S. regulators.

Maria Jose Contreras shared the story of her two sons Franco, 9, and Julian 6, having had a rare genetic disease with doctors informing her that they were “unlikely to survive to see their 30^th birthday.”

She volunteered her sons to participate in an experimental gene therapy trial which ultimately reversed symptoms of the disease including weakening muscles.

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“The therapy stopped the disease from progressing. Now, they can walk for longer periods of time without getting tired and Franco enjoys playing football with his classmates” Contreras said.

This gene therapy (SRP-9001) is being considered as a new treatment option for Duchenne muscular dystrophy, a muscle-weakening disease that affects one in 3,500 boys and a smaller number of girls.

The Food and Drug Administration is expecting to decide on its approval by today; however, some health experts warn that there is not enough evidence the therapy works.

The FDA is working on an accelerated approval process designed to give the greenlight to new therapies for serious conditions without initial human trials needing to be completed and approved.

Critics argue that although the company asked the agency to approve the drug for Duchenne patients in every age group, without providing the scientific evidence, the accelerated approval process would potentially reach some trouble after green-lighting a controversial Alzheimer’s drug in 2021.

The SRP-9001 gene would be inserted inside the muscle cells which could carry instructions for them to begin formulating a smaller version of a protein called micro-dystrophin.

Sarepta and some scientific experts say this protein improves muscle and organ function in patients which may improve a quality of life and lifespan.

According to the article, however, “there is limited scientific data showing the therapy’s clinical benefit with just the first phase of a randomized trial over 48 weeks completed by Sarepta.”