For weeks there has been speculation, guesses, and hope about what we might learn. We have been following the stories on social media, grateful for every photo or video. We are thrilled that the open label eteplirsen studies (confirmatory study and non ambulatory study) are actively recruiting and are anxiously awaiting the timeline for follow-on exons.
For years we have suggested the benefit/risk equation is different in Duchenne than in other diseases and PPMD's benefit/risk pilot confirmed and quantified the equation. Parents highest priority – second only to a cure – is to slow disease progression.
The 168 week data exceeds our expectations. Eteplirsen slows the rate of progression, delays the loss of ambulation, and preserves respiratory function.
We must keep in mind that the brave young men in the study, all 12 of them, were 9+/- years of age when starting the study. When entering the study, their muscles had already been damaged because dystrophin was absent, resulting in an unhealthy cellular environment. The dystrophin that was confirmed after 48 weeks has led to this long-term benefit through 168 weeks. After 168 weeks, their rate of decline was far less than predicted.
Slowing degeneration, preserving ambulation, stabilizing respiratory function preserve the quality of life for these young men, helping to maintain the integrity of the spine and delay the need for non invasive ventilation.
We all recognize that it will take a combination of drugs to stop progression, that doctors will need an arsenal of tools to deliver precision medicines for every individual diagnosed. Eteplirsen meets the criteria as an effective drug to slow progression.
As a community we fully support eteplirsen as a therapy. We completely understand that treating people with Duchenne earlier will likely provide additional benefits. We also agree that slowing progression for all individuals, no matter the age or stage, is critically important and we support all efforts to fully exploit this technology.
We are and continue to be believers.