Company: SRPT
Filing Date: 2025-02-28
Form Type: 10-K
Source: 0000950170-25-029973
Chunk: 154

Company: Sarepta Therapeutics, Inc.
Filing Date: 2025-02-28
Form: 10-K
Item: Item 1
Chunk 154
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 or a “Regenerative Medicine Advanced Therapy (“RMAT”)” designated product, or may seek approval through the accelerated approval pathway or under priority review.

•Fast Track Designation: Fast track products are those products intended for the treatment of a serious or life-threatening disease or condition and which demonstrate the potential to address unmet medical needs for such disease or condition. If fast track designation is obtained, the FDA may initiate early and frequent communication and begin reviewing sections of a marketing application before the application is complete. This “rolling review” is available if the applicant provides, and the FDA approves, a schedule for the remaining information. 

•Breakthrough Therapy Designation: Breakthrough therapy designation is focused on expediting the development and review process and by itself does not create an alternate ground for product approval. A sponsor may seek FDA designation of a drug candidate as a breakthrough therapy if the drug is intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. The FDA issued guidance entitled “Expedited Programs for Serious Conditions––Drugs and Biologics” in May 2014.

•RMAT Designation: RMAT designation may be granted to drug products that meet the statutory definition of RMAT; are intended to treat, modify, reverse, or cure a serious condition; and for which preliminary clinical evidence indicates that the RMAT has the potential to address unmet clinical needs for such condition. The statutory definition of an RMAT includes therapies such as our gene therapy product candidates.

•Accelerated Approval: the FDA may also approve products through the accelerated approval pathway, which is aimed at expediting review of drugs that treat serious conditions and provide a meaningful advantage over available therapies. Accelerated approval is based on demonstrated effect on a surrogate endpoint that is reasonably likely to predict clinical benefit or on a clinical endpoint that can be measured earlier than irreversible morbidity or mortality (“IMM”) that is reasonably likely to predict an effect on IMM or other clinical benefit (i.e., an intermediate clinical endpoint). Approvals of this kind typically include requirements for appropriate post-approval Phase 4 clinical trials to confirm clinical benefit. 

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The Food and Drug Omnibus Reform Act of 2022 (“FDORA”) signed by former President Biden on December 29, 2022 as part of the Consolidated Appropriations Act, 202