Sarepta Therapeutics has partnered with Idis Managed Access, a division of Clinigen Group, to develop a managed access program (MAP) for its lead therapy Exondys 51 (eteplirsen) for the treatment of Duchenne muscular dystrophy (DMD).
The MAP was planned to provide early, expanded access to Sarepta’s Exondys 51 to patients who have mutations in the dystrophin gene that can be corrected by elimination of a part of the gene (exon 51).
The program will provide legal and ethical mechanisms to clinicians so they can prescribe Exondys 51 to patients who meet the pre-specified medical criteria, and who can ensure the necessary financial support.
DMD is caused by mutations in the gene that encodes for the protein dystrophin — an important protein for normal muscle function.
Exondys 51 therapeutic activity is based on a gene regulatory mechanism called exon skipping, in which parts of genes called exons are cut off, giving rise to proteins that are smaller than the original ones. Exondys 51 takes advantage of this process; it binds to the dystrophin gene and causes its exon 51 to be skipped. This will produce a new and functional dystrophin protein.
Because Exondys 51 was specifically designed to target the exon 51 of the gene, this therapy is only suitable for patients who carry mutations in this specific part of the gene, which occurs in about 13 percent of DMD patients.
Clinical data from a small number of DMD patients treated with Exondys 51 have showed that it is safe and well tolerated. However, the long-term safety and therapeutic effectiveness of Exondys 51 have not been established.
Exondys 51 received accelerated approval in September 2016 by the U.S. Food and Drug Administration (FDA), but additional clinical testing is still required before the FDA issues a full approval. This means DMD patients amenable to exon 51 skipping may have access to the treatment in the U.S., but it is unavailable in many other countries.
For now, the MAP will be accessible to patients who live in Argentina, Brazil, Canada, Colombia, France, Germany, Greece, Iceland, Italy, Mexico, Spain, Turkey, and the United Kingdom.
Sarepta expects to expand the list of countries covered by the program in the future. All requests must be filled out by the treating physician on behalf of the patient.
More details on the Exondys 51 Managed Access Program can be requested by phone to a Clinigen representative (+44-1283-494-340) or by e-mail to [email protected].