Treatment Access

Sarepta is committed to developing innovative, safe and effective precision genetic medicines to treat serious diseases, many of which are fatal with limited or no treatment options. We take our commitment to the patients we serve very seriously, with shared urgency to develop new treatments as quickly as science and regulatory requirements allow. We believe the best way to fulfill this responsibility is to conduct clinical trials to assess safety and effectiveness and obtain regulatory approval so that our therapies reach as many patients as possible.

It is our goal to design inclusive clinical trials with broad patient eligibility when doing so will not compromise the scientific validity of the trial and its findings in support of regulatory approval. Designing rare disease clinical trials can present unique challenges, often because the disease can look very different from one person to the next, depending on age, disease stage, and other factors. In order to determine whether a treatment works or not, it is important to minimize variability that could be introduced by these characteristics when designing clinical trials. This means it can be scientifically and clinically difficult to enroll a broad patient population in a given study and there may not be a trial fit for every patient.

In light of this, we understand the interest in access to our investigational (pre-approval) therapies outside of the clinical trial setting through compassionate use which includes the Expanded Access or Right To Try pathways.* Use of these pathways for pre-approval access to our investigational therapies must balance our shared urgency with many other factors. These include regulatory and practical considerations, such as each agent’s benefit-risk profile, available supply, impact on clinical development, feasibility of providing fair and sustainable access, and availability of a clear path to regulatory approval.

At this time, we are unable to offer a compassionate use program for any of our investigational exon skipping or gene therapies without jeopardizing our ability to conduct the trials needed to gain timely regulatory approval and provide patients broad, equitable, and sustainable access. We believe the most appropriate way to access our investigational therapies is to participate in clinical trials regulated by the Food and Drug Administration (FDA) and other regulatory entities across the globe.  Executing our current and future clinical trials as thoughtfully and efficiently as possible remains the best path to potential regulatory approval and ensuring broad availability of our therapies.

We will continue to evaluate the possibility of compassionate use and will update our policy if our position changes. Should patients or their families have further questions, or require additional information about the status of our investigational therapies, please contact Sarepta Patient Affairs by email at [email protected]. Physicians should contact Sarepta Medical Affairs by email at [email protected] or in the United States toll free at 1-888-727-3782, option 2.  We anticipate acknowledging receipt of requests sent by email within 5 business days.

*Compassionate use refers to requests for access under the Federal Food, Drug, and Cosmetic Act Section 561A (FDA’s Expanded Access program) and Section 561B (“Investigational Drugs For Use By Eligible Patients” as added by the Right To Try Act, P.L. 115-176)