EXONDYS 51 is the brand name for eteplirsen is the first treatment approved by the Food and Drug Administration (FDA) on September 19th, 2016 for a specific group of patients with Duchenne muscular dystrophy (DMD).
Frequently Asked Questions About EXONDYS 51TM
Is my son eligible for EXONDYS 51TM?
EXONDYS 51TM is for patients with a confirmed genetic mutation that is amenable to exon 51 skipping. For exon skipping to work, exon 51 must be present.
How does EXONDYS 51TM work?
EXONDYS 51TM is designed to bind exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing.
How is EXONDYS 51TM given to patients?
The exon-skipping therapy is given intravenously (through the veins) as a once-weekly injection.
What is the benefit of EXONDYS 51TM?
In clinical trials, EXONDYS 51TM increased dystrophin in the muscles of some patients. It is believed that this increase may predict clinical benefit in patients. More trials are ongoing to assess whether there is a clinical benefit.
Is EXONDYS 51TM covered by insurance?
Insurance coverage varies from plan to plan and it will require prior authorization. Depending on your insurance review process, prior authorization rate of acquisition may vary. A patient assistance program for those using EXONDYS 51TM , called SareptAssist, is available to help with treatment use and availability, and insurance and other financial concerns.