Rare Disease Report

Exondys 51 Will Insurance Companies Cover this Duchenne Drug?

JUNE 23, 2017
James Radke
An article published in the New York Times this week showcases the problems many Duchenne muscular dystrophy (DMD) patients are having in acquiring access to the recently approved Exondys 51 (eteplirsen).
DMD, a progressive, degenerative muscle disorder, is caused by a lack of functional dystrophin, and symptoms typically begin in early childhood. By the time a boy is in his early teens, he is often wheelchair bound. As the disease progresses, the patient’s muscles will continue to deteriorate and most will die in their 20s.
Exondys 51 treats boys with DMD amenable to exon 51 skipping therapy.
The primary issue that payors have regarding the drug is that it was approved based on data that was very conclusive based on the patients’ testimonials, but somewhat questionable from a statistically perspective. The drug was given an Accelerated Approval by the FDA last year, which means the company developing it, Sarepta, is currently conducting a Phase 3 study to confirm that the drug is effective.
The accelerated approval along with the statistical uncertainty of the drug's efficacy, means that while the drug is approved, some insurance companies would prefer to wait until more data is available before paying for it.
In the New York Times video below, Michelle Lessner-Gonzales, the mother of 14-year-old DMD patient Nicolas, shows her concerns about the limbo she is in while waiting for the Illinois Medicaid program to decide how it will cover Exondys 51.

Her frustration comes mostly because DMD is a progressive disease and, as Nicholas, and other DMD boys continue to wait for the drug, their bodies will continue to deteriorate. In a classic ‘Catch-22’ problem, if the decisions get delayed to the point where Nicholas is no longer able to walk, he may not qualify for the drug, based on some insurance companies’ criteria that patients be ambulatory to receive the drug. (see RDR’s coverage of that problem here and here).
When Exondys 51 was approved last fall, the entire Duchenne community rejoiced. The celebration has been replaced by frustration and concern.
Christine McSherry of the Jett Foundation, who was heavily involved in providing the FDA with patients’ testimonials at the FDA review of the drug, noted that she is now reading  coverage denial letters.
McSherry said, “It’s very disheartening to have worked that hard, and to have sacrificed that much, and to now have to battle the insurance companies.”
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