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FDA Converts Sarepta Therapeutics DMD Gene Therapy to Full Approval, Expands Indication
While some review teams had wanted to issue a complete response letter, CBER Director Peter Marks overruled the recommendation, per FDA documents.
Sarepta Therapeutics' Bid to Expand DMD Gene Therapy Indication Under FDA Priority Review
Sarepta is hoping to expand the indication for Elevidys to all ages and convert its accelerated approval to full approval.
Sarepta Defeats Gene Therapy Patent Infringement Claims From Regenxbio, UPenn
Sarepta had been accused of using methods patented by UPenn and licensed by Regenxbio to create its DMD gene therapy.
Sarepta Therapeutics Submits New Data to FDA to Expand Elevidys Indication, Convert to Full Approval
The agency in June granted the firm accelerated approval for Elevidys, the first gene therapy for Duchenne muscular dystrophy in the US.
Sarepta Focused on Expanding DMD Gene Therapy Label Despite Falling Short on Primary Endpoint
Despite not meeting the primary endpoint in the EMBARK trial, the company remains motivated to seek a label expansion from the FDA based on secondary endpoints.