The FDA’s accelerated approval of a gene therapy for patients aged 4 to 5 years old is an important milestone for the Duchenne community but the work towards identifying truly transformative treatments is far from done, as Debra Miller, the CEO of CureDuchenne, explains

By Debra Miller, CEO of CureDuchenne

We are pleased that the FDA has granted accelerated approval of SRP-9001, as it is an important milestone that keeps us on a path toward identifying truly transformative treatments for those living with Duchenne muscular dystrophy.

 While the FDA has granted this approval for patients aged 4 to 5 years old, we recognise that many in the community will be disappointed due to the narrow age range specified. We look forward to the full data readout from the EMBARK trial and expect this confirmatory study will generate important data to further inform the best clinical use of this therapy. We are hopeful it will demonstrate efficacy and safety to support an expanded age range, and eventually expand to include non-ambulatory individuals.

While this is a significant milestone, our work is far from done. There are still many individuals waiting for effective treatments, and CureDuchenne will continue to support and invest in a diverse array of promising research to bring solutions to everyone living with Duchenne, including many technologies that could potentially overcome the limitations of first-generation gene therapies.

cureduchenne.org

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