PPMD is deeply disappointed that Dr. Peter Marks has made the decision to leave his role as Director of the FDA’s Center for Biologics Evaluation and Research (CBER). In his resignation letter, addressed to Acting FDA Commissioner Sarah Brenner, Dr. Marks indicated that his departure will be effective April 5, 2025. This leadership change is profoundly unsettling for the rare disease community and all who rely on a stable, expert-driven regulatory environment.

Dr. Marks has been an extraordinary public servant and a steadfast advocate for individuals living with rare diseases, including Duchenne and Becker. Under his leadership, CBER prioritized innovation and meaningful patient engagement. Dr. Marks recognized that the voices of patients and families are central to regulatory decisions, and he worked tirelessly to ensure those voices informed and shaped the path forward. His legacy is one of courage, compassion, and commitment to improving lives.

We are deeply grateful for Dr. Marks’ years of leadership and unwavering dedication to the rare disease community. His departure underscores the need for continued, unified advocacy. Now more than ever, we urge the administration to trust in data, uphold scientific integrity, and maintain critical investments in drug development and research. Our community cannot afford delays or disruptions in access to safe, effective therapies.

PPMD remains steadfast in our commitment to protecting and advancing the progress we’ve fought so hard to achieve. We are committed to working closely with regulatory experts, policymakers, and partners to understand the full implications of this personnel change. We urge the administration to prioritize the needs of the rare disease community by maintaining strong, experienced leadership at the FDA and ensuring a continued commitment to regulatory pathways that support timely and safe access to new therapies.

While PPMD remains committed to advocating for our community and working tirelessly to resolve this issue as quickly as possible, today, we focus on thanking Dr. Marks for his leadership and service to the Duchenne and Becker community.