Treatments of Interest:

  • deflazacort (Emflaza®, PTC Therapeutics)
  • eteplirsen (Exondys 51®, Sarepta)
  • golodirsen (Vyondys 53®, Sarepta)

Duchenne muscular dystrophy (DMD) is a genetically inherited neuromuscular disease that almost entirely affects boys and results in a progressive loss of muscle function, resulting in progressive weakness and eventual death usually from cardiac and respiratory failure. It is the most common pediatric muscular dystrophy with a prevalence of one in 3,500 – 5,000 live male births, or about 400 to 600 boys per year in the US.

The public meeting revealed tensions inherent around high-priced treatments that lack adequate evidence of efficacy.

Eteplirsen was approved by the FDA in September 2016 for patients with a confirmed mutation in the dystrophin gene that is amenable to exon 51 skipping. Sarepta has announced its intention to file for accelerated approval of golodirsen by the end of 2018.

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Final Documents

Below you will find the final documents from the assessment review process:

“DMD is a devastating disease. Parent testimony shared throughout the day provided a window into the hope and frustration that families are feeling as new treatments become available. In the case of deflazacort, we heard about how an inexpensive drug that was available overseas for years and could be easily brought into the US became unaffordably expensive with FDA approval when the US manufacturer massively increased its price. With eteplirsen, we heard about the hopes of parents that the treatment is beneficial. However, the wholly inadequate evidence base from the manufacturer has created such doubt that payers, faced with the outrageously high price charged by the manufacturer, have created extremely narrow coverage policies; no other country has even approved the treatment. We are left where no one can know whether a useful therapy is being administered to a small number of children while others around the world are denied therapy by payer barriers and regulatory approval, or whether the patients receiving eteplirsen are being given a useless, high-priced treatment. Three years after approval, the manufacturer has provided no high-quality evidence of benefit, many patients and families are surely losing out, and only the manufacturer truly benefits from this deplorable situation. Families and patients with DMD deserve better.”