Sarepta is committed to developing innovative, safe and effective therapies for patients with Duchenne muscular dystrophy (DMD). We take our responsibility to act as a true partner with the Duchenne community very seriously, and are committed to ensuring our innovations reach as many patients as possible, as quickly as possible.
We understand that patients and families are interested in accessing our DMD exon-skipping investigational therapies prior to regulatory approval, and also outside of the clinical trial setting, through compassionate use programs.* Compassionate use and other mechanisms for pre-approval access to our DMD exon-skipping agents must balance our shared urgency with many other factors. These include regulatory and practical considerations, such as each agent’s benefit/risk profile, the impact on clinical development, and the feasibility of providing fair and sustainable access.
After much consideration, we have determined that, at this point in time, we are unable to offer a compassionate use program on a fair and sustainable basis for any of our investigational agents without jeopardizing our ability to provide patients broad, sustainable, and long-term access. We believe that the best path to potential regulatory approval, and subsequent access to the greatest number of patients, is by executing our current and future clinical trials as efficiently as possible.
We will continue to evaluate the possibility of compassionate use and pre-approval access mechanisms. We will update the Duchenne community if any such programs can be conducted in a fair and sustainable way without compromising clinical development and potential regulatory approval of any of our DMD exon-skipping investigational therapies.
Should patients, families, or physicians have further questions or require additional information about the status of our exon skipping therapies, please contact our Global Medical Information department by email at firstname.lastname@example.org or in the United States toll free at 1-888-727-3782, option 2.
*Compassionate use programs include requests for access under section 561(b) of the Federal Food, Drug, and Cosmetic Act, as well as ‘Right to Try’ legislation.