With the accelerated approval of EXONDYS 51™ (aka eteplirsen) by the FDA in September, the Duchenne community has been quickly introduced to a whole new vocabulary and a whole new list of concerns. We knew that achieving an accelerated approval was going to be difficult. But the process of watching policy determinations and caveats to access be administered from health insurers unfamiliar with our Duchenne community and this product presents all new layers of uncertainty and worries that feel cruel and unethical.


These days, most of us expect challenges from our insurance companies – especially when you have been navigating insurance coverage and Duchenne. For the last few years, PPMD, in anticipation of the day we would get an approval(s), has been trying to better understand the access and reimbursement environment.

What we have learned is that every company is different. And no decision is final or set in stone. This is a process.


Decisions Aren’t Final


Soon after the FDA’s Accelerated Approval of EXONDYS 51, Anthem announced that they would not be covering the therapy because they deemed it to be ”investigational”. PPMD sent Anthem a letter and to date, have not received a response. 


Other companies appear to be supporting EXONDYS 51, but with limitations and restrictions. PPMD is engaging with many of these insurance providers in an effort to ensure that access to EXONDYS 51 is as broadly available as was intended by the FDA’s labeling (which imposed no age or gender restrictions). PPMD has also been in touch with Sarepta and they are working closely with all major health insurers.

Again, the decisions made to date are not final. This is an intense process and we are hopeful that as Sarepta meets with the various health insurance providers, these providers will have a better understanding of the importance of this therapy to our community. We highly recommend that you visit SareptAssist.com or call 1.888.SAREPTA to find out if your insurance provider covers this therapy.


What can you do?


We know how frustrating this process is. There are a few actions you can take to try to encourage your provider to cover EXONDYS 51.

  1. First, make sure you are amenable to exon 51 skipping and EXONDYS 51. Get your free genetic testing, interpretation, and counseling through Decode Duchenne: visit DuchenneConnect.org/DecodeDuchenne.
  2. Contact SareptAssist at SareptAssist.com or call 1.888.SAREPTA. SareptAssist has a team of Case Managers ready to help you navigate all of the logistics that accompany access to EXONDYS 51.
  3. As we all know, direct advocacy to your health insurance provider is always beneficial.  In consultation with your physician, establishing a relationship with the Case Manager at your insurance company is recommended.  To identify your Case Manager, call the number on the back of your medical insurance card.

What if I am not amenable to exon skipping or EXONDYS 51?


While only a percentage of our community is treated by this first therapy, as more drugs are approved that treat Duchenne, we will all be battling similar issues. We encourage you to stay connected with PPMD as we continue to bring you updates on our Duchenne community’s access environment. Like so many things in the fight to end Duchenne, knowledge is power and staying engaged can help arm you with information that may not make a difference today…but could help down the road.


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