Top 5 FAQs: Eteplirsen (Exondys 51) for DMD Treatment

On Sept. 19, the U.S. Food and Drug Administration granted accelerated approval to eteplirsen, which will now be marketed under the brand name Exondys 51 for the treatment of some forms of Duchenne muscular dystrophy (DMD).

We’ve received a lot of questions about the newly approved drug, and wanted to share a few answers to the most common. (Be sure to see the note at the end of this blog about an upcoming webinar where we’ll be answering more in-depth questions):

1. Can Exondys 51 be used to treat all forms of DMD or only certain types?

Exondys 51 is an exon skipping drug that specifically targets a section of genetic code called exon 51 in the dystrophin gene. It’s estimated that 13 percent of boys with DMD could benefit from skipping exon 51.

Additional exon skipping drugs that could impact other forms of DMD are moving through the drug development pipeline. At this time, researchers currently are working on development of exon skipping drugs to target exons 44, 45, 50, 52, 53 and 55, as well as strategies to target multiple exons. DMD therapies based on other strategies are under development as well.

2. Is there a specific age range for which Exondys 51 will work?

The clinical trials that supported approval of the drug were conducted in boys ages 7-13 years (at the time the studies started). Sarepta Therapeutics currently is conducting a number of clinical trials to determine the efficacy of Exondys 51 in young boys and men ages 4-21 years. The best thing to do at this time is to speak with your doctor about what may be best for you or your child.

3. What will treatment with Exondys 51 cost, and will insurance cover it?

Details haven’t yet been released about insurance and out-of-pocket costs, so we have no way of knowing what those costs will be until the drug becomes available on the market. We will keep the community informed as we learn more. In the meantime, Sarepta Therapuetics has set up the SareptAssist Patient Support and Assistance program, through which case managers are available to help navigate the complexities of beginning a therapy.

4. Can Exondys 51 help other types of muscular dystrophy?

Although this drug is designed to treat certain forms of DMD, we’re funding a lot of cutting-edge research aimed at finding treatments and cures for all the neuromuscular diseases in our program. Search for a particular disease on our site or visit our Grants at a Glance page to learn more about the projects we’re funding across the United States and around the world.

5. Will Exondys 51 be made available in other countries?

There is no information at this time about whether Sarepta Therapeutics will seek approval to market this drug in countries outside the U.S., but we will post updates on any new developments as we learn more.

Have more questions about Exondys 51? On Thursday, Oct. 6, we’ll be co-hosting a webinar with Sarepta Therapeutics specifically for DMD families where you’ll have the opportunity to ask more in-depth questions related to Exondys 51. Be sure to like our Facebook page, as we’ll be releasing webinar sign up information there soon.