Sarepta Therapeutics says the US FDA needs additional time to review Duchenne muscular dystrophy (DMD) gene therapy delandistrogene moxeparvovec (SRP-9001).
In November 2022, the US Food and Drug Administration (FDA) accepted the submission from Sarepta for its potential DMD gene therapy SRP-9001. The Agency initially provided the firm with an action date of May 29, 2023; however, this has now been delayed until June 22, 2023.
The additional time requested by the FDA to complete the review will also include post marketing commitment discussions and label negotiations.
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Furthermore, subject to the competition of the Biologics License Application (BLA), the FDA indicated it could initially approve the gene therapy for use in patients ranging from four to five years old. Since the agency delayed an accelerated approval decision and implied it would first be authorized for a smaller size patient group, Sarepta’s shares have decreased by 7%.
SRP-9001 is in Phase III trials and the FDA told Sarepta it needs to confirm the results of the initial BLA approval and if the study meets its objectives the organization will then “entertain” a non-age-restricted expansion of the DMD gene therapy based upon the data available.
Sarepta said it will remain in a quiet period for the duration of the BLA review.
For its gene therapy pipeline, Sarepta had been using Paragon Bioservices for viral vector supply, entering into a specific long-term manufacturing agreement for SRP-9001 in October 2018. A year later, contract development manufacturing organization (CDMO) Catalent acquired Paragon for $1.2 billion and rapidly the agreement to add a second dedicated production line for Sarepta at its site in Harmans, Anne Arundel County, Maryland.
In January, Sarepta announced it had expanded its DMD gene therapy deal with Catalent, in which the CDMO will supply commercial doses of SRP-9001 to the firm once it wins FDA approval.
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