gene therapy

FDA grants approval via accelerated approval pathway to ELEVIDYS: First-ever gene therapy for individuals with Duchenne ages 4-5

Today is an incredible milestone for the entire Duchenne community. It is a first step. But a huge step forward. Today, the FDA has granted accelerated approval to ELEVIDYS (delandistrogene moxeparvovec-rokl), a micro-dystrophin gene therapy…

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Sarepta Announces PDUFA Extension and Update for the FDA’s Review of SRP-9001

Sarepta Therapeutics announced today that they received an update from the FDA on the Biologics License Application (BLA) for SRP-9001. The PDUFA date reportedly has been extended to June 22, 2023. …

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SRP-9001: Submit Your Community Perspectives to FDA

On May 12, the FDA held an Advisory Committee meeting for SRP-9001, a gene therapy intended to treat Duchenne. The Advisory Committee meeting was used by FDA to obtain independent expert advice on scientific, technical,…

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A Community United: Thank You for Raising Your Voice During the SRP-9001 Advisory Committee

Today was a historic day for the Duchenne community: the first FDA Advisory Committee meeting for a gene therapy intended to treat Duchenne. This meeting was held as part of the regulatory review process for…

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May 12: Advisory Committee Meeting for SRP-9001 – Live Stream Information & Meeting Materials Released

The FDA has shared live stream access information for the May 12th Advisory Committee for SRP-9001 (delandistrogene moxeparvovec), Sarepta Therapeutics’s investigational gene therapy, which has applied for FDA approval through the Accelerated Approval pathway with…

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What You Need To Know Ahead of the SRP-9001 Advisory Committee on Friday, May 12

This Friday, May 12th, will be a historic day for the Duchenne community. It will be the day of the first ever FDA Advisory Committee meeting for a gene therapy specifically to treat Duchenne. PPMD…

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PPMD Submits Findings from New Gene Therapy Preference Study to FDA for SRP-9001 Advisory Committee

May 12 will be a historic day for the Duchenne community: the first FDA Advisory Committee meeting for a gene therapy specifically intended to treat Duchenne. PPMD, our community partners, and many others have long…

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PPMD & Community Partners Submit Letter to FDA Encouraging Appropriate Regulatory Flexibility in SRP-9001 Review

PPMD along with community partners and key opinion leaders and physicians in the Duchenne space have submitted a letter to the US Food and Drug Administration (FDA) and FDA’s Center for Biologics Evaluation and Research…

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Pfizer Completes Enrollment of CIFFERO Phase 3 and DAYLIGHT Phase 2 Gene Therapy Trials

Pfizer announced today that enrollment is now complete for both the Phase 3 CIFFERO study and Phase 2 DAYLIGHT study of PF-06939926 (fordadistrogene movaparvovec) in Duchenne. As always, we are so grateful to all of…

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How to Submit Written Testimony for the SRP-9001 FDA Advisory Committee Meeting – Deadline May 5th

Sarepta recently announced that the FDA Advisory Committee meeting for SRP-9001 (delandistrogene moxeparvovec), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, will be held on May 12th. The event will be completely virtual and a…

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