GENERAL OVERVIEW OF MANAGED ACCESS PROGRAM
Sarepta is committed to developing innovative therapies for patients with Duchenne muscular dystrophy (DMD). We take our responsibility to act as a true partner with patient communities seriously, and we are committed to working with physicians who have determined that their patient has an unmet medical need.
We believe that well-designed clinical trials are the best way to determine the safety and effectiveness of investigational therapies and for patients to gain access prior to regulatory approval. However, we understand that patients and families may be interested in accessing our investigational therapies prior to regulatory approval, and outside of the clinical trial setting, through managed access programs (also known as an early / expanded access, or named patient program).
Managed access programs provide a mechanism through which physicians can legally and ethically prescribe an unapproved therapy to patients who meet pre-specified medical criteria and can secure funding.
MANAGED ACCESS PROGRAM ELIGIBILITY FOR ETEPLIRSEN
For certain patients in certain jurisdictions, as part of our commitment to facilitating patient access, we support a limited managed access program in selected geographies where eteplirsen is not currently approved. We plan to expand the program to include more countries over time.
COUNTRIES WHERE THE PROGRAM IS OFFERED
Argentina, Brazil, Canada, Colombia, France, Germany, Greece, Iceland, Italy, Mexico, Spain, Turkey, and United Kingdom.
All requests must be submitted by the treating physician on behalf of the patient.
HOW DO I SUBMIT A REQUEST FOR MANAGED ACCESS?
Healthcare providers can obtain details about submitting requests by calling a Clinigen representative at +44 1283 494 340, or emailing firstname.lastname@example.org.
CLINICAL DEVELOPMENT PROGRAMS
At this time, we are unable to provide managed access or compassionate use programs for our other investigational therapies or additional treatment modalities currently in clinical development. We will continue to evaluate the possibility of compassionate use and pre-approval access mechanisms in the future and will institute such programs should it be feasible to conduct such a program in a fair and sustainable way without compromising clinical development and / or potential regulatory approval of any of our investigational therapies.