Defeat Duchenne Canada is disheartened to share the news that Pfizer has decided to discontinue development of their fordadistrogene movaparvovec gene therapy program for Duchenne muscular dystrophy (DMD). This comes after the announcement in June of the results of their Phase 3 CIFFREO study that did not meet its primary endpoint of improvement in motor function among ambulatory boys 4 to 7 years of age treated with this gene therapy compared to placebo, nor its key secondary endpoints.

While the development program has discontinued, Pfizer will continue with the long-term study to ensure the safety of all participants in the trial, including those trial sites in Canada. Pfizer will also continue to provide data from the long-term safety study through medical and patient advocacy forums. We are hopeful that the resulting data will help to support future research into DMD, and potentially inform other gene therapies that are under development in the area.

We are aware of how difficult this news is, especially for those who participated in the trial and their loved ones. Please reach out to us with any questions or concerns you might have about this difficult news. Please see Pfizer’s community letter below.

Want to know more about Duchenne Research?

Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: