The month-long series of good news for the Duchenne community has come to an end. Medical Insurance company, Anthem Inc. has issued a statement
saying they will not cover Sarepta's recently approved Exondys 51 (eteplirsen) for the indication it was given (for Duchenne boys amenable to exon 51 skipping therapy). Anthem stated that Exondys 51 is still investigational and not medically necessary for patients.
Anthem is the United States’ second largest health insurer.
Last month, the FDA granted accelerated approval of Exondys 51 with the condition that a final phase 3 clinical trial be conducted. Anthem, in turn, questions the FDA's approval and sites the FDA’s own documents from earlier this year which were very critical of the data.
Whether or not other insurance companies follow suit remains to be seen but as it is now, any families seeking treatment with Exondys 51 and insured by Anthem will have to find another means of being able to pay for the drug.
It is estimated that the drug costs an average of $300,000 per year but that will vary based on the child’s size.
No comment from Sarepta has been made yet.
Duchenne muscular dystrophy
Duchenne muscular dystrophy is caused by lack of a functional dystrophin protein, a protein that helps keep muscle cells intact. Patients with progressive muscle disorder experience symptoms in early childhood, losing the ability to walk as early as age 10. These patients, mostly boys, experience life-threatening heart and lung complications in their late teens and twenties.
There are many subsets of the Duchenne population based on the type of mutation found in the dystrophin gene. There are currently no drugs approved in the US to directly treat any of these groups.
Exondys 51 is designed to enable RNA to skip over the part of the DNA with a problematic mutation, enabling a functional (though shorter) dystrophin protein to be produced. Specifically, the drug skips a portion of the DNA known as exon 51. About 13% of the 35,000 DMD patients in the US and Europe have a mutation that might theoretically respond to a drug such as Exondys 51.