PPMD acknowledges the media reports that the U.S. Food and Drug Administration (FDA) will request Sarepta Therapeutics stop shipping ELEVIDYS for all Duchenne patients. These reports are profoundly upsetting and raise serious concerns for our entire community. Families who have fought tirelessly for access to this therapy, those who have already received it, and those who are in line to receive it are now left with more questions than answers.
ELEVIDYS was the first gene therapy approved for Duchenne and set an important precedent for the development and evaluation of future therapies. Its approval signaled both scientific and regulatory readiness to explore genetic approaches to treating Duchenne and underscored the importance of continued investment and collaboration in the rare disease space.
PPMD is urgently seeking answers and calling on both Sarepta and the FDA to provide clarity and transparency. Above all, we will remain steadfast in our commitment to the Duchenne community, to protect access, demand transparency, advocate for science-driven, patient-centered regulatory decisions, and ensure that individuals and families receive the answers and support they need.
PPMD is pulling together expert clinicians and community leaders to discuss next steps and assess the implications of this potential withdrawal and determine the best path forward for families. We recognize the urgency and anxiety this announcement has created. Our expert partners will help us evaluate the medical, regulatory, and practical consequences of this development and ensure families receive accurate information and guidance. PPMD is committed to providing timely updates and actionable resources as this situation unfolds.
PPMD remains unwavering in our commitment to the Duchenne community. To every parent, patient, and caregiver: you are not alone. PPMD is mobilizing the full strength of our network, from clinical experts to legal advisors to get answers. Together, we will navigate this, as we always have, with resilience and urgency, protecting the safety of our community while continuing to drive forward the momentum of life-altering innovations. PPMD will continue to monitor this evolving situation closely and will provide timely updates to the community as new information becomes available.
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