Research
Treatments
The National MPS Society expresses deep appreciation and concern following the announcement that Dr. Peter Marks will resign from his role as Director of the U.S. Food and Drug Administration’s Center for Biologics Evaluation and Research (CBER). In a letter to the FDA Dr. Marks stated that his departure will be effective April 5, 2025.
This leadership change is a significant loss for the rare disease and MPS communities, and it comes as a significant blow to all who rely on stable, expert-driven regulatory oversight. Dr. Marks has been an invaluable champion for patients with Mucopolysaccharidoses (MPS), and his resignation leaves a profound void in the effort to develop and approve life-saving therapies for rare diseases.
During his nine-year tenure leading CBER, Dr. Marks oversaw the approval of 22 novel gene and cell therapies – groundbreaking treatments that have brought hope to families facing conditions once thought untreatable.
Dr. Marks’ personal commitment to the MPS community was evident in his direct engagement with patients and families. He delivered a keynote address at the National MPS Society’s 37th Annual Family Conference in 2023 in a session aptly titled “Addressing the Unmet Needs of the Rare Disease Community.” At this event, Dr. Marks spoke candidly and empathetically about the challenges of developing therapies for ultra-rare diseases. He exhibited a deep understanding of and sympathy towards MPS families in his comments during the panel session.
By taking the time to engage at the conference, Dr. Marks demonstrated extraordinary commitment to those living with MPS, reinforcing the principle that patients and caregivers must be partners in the drug development process. He recognized that the voices of patients and their families are central to regulatory decisions, and worked relentlessly to ensure those voices informed and shaped the path of drug development. His leadership fostered innovative gene therapy programs while instilling a culture of compassion in the regulatory process, directly benefiting the MPS community and beyond.
Moving Forward – Protecting Progress and Calling for Continued Leadership:
Dr. Marks’ resignation comes at a pivotal moment for rare disease research and regulation. The National MPS Society reaffirms our commitment to protect the progress we have found so hard to achieve in rare disease drug development. We will continue to work tirelessly with policymakers, regulators, and industry partners to ensure that the momentum in developing therapies for MPS and ML remains uninterrupted.
Our community cannot afford delays or disruptions. We urge the FDA and the Administration to maintain strong, experienced leadership at the Agency to support the needs of patients with rare diseases, and we stand ready to collaborate with FDA leadership to sustain the uninterrupted momentum in rare disease therapy development so that the innovative treatments on the horizon become realities for the families who need them the most.
About the National MPS Society
Founded in 1974, the National MPS Society is a 501(c)3 non-profit organization dedicated to supporting individuals and families affected by Mucopolysaccharidoses and Mucolipidoses. The Society’s mission is to advance research for cures, provide advocacy and support, and promote awareness of these devastating, progressive diseases.
Contact: Terri Klein, President and CEO
terri@mpssociety.org
