CEP Patient-Focused Drug Development Meeting
In an important milestone for the CEP community and rare disease advocacy, United Porphyrias Association hosted an Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting on Congenital Erythropoietic Porphyria (CEP) on October 14, 2025.
Patient panelists at the CEP EL-PFDD meeting on October 14, 2025.
The goal of this meeting was to provide the U.S. Food and Drug Administration (FDA), product developers, clinicians, and academic researchers an opportunity to hear perspectives from individuals with CEP on the health effects and daily impacts of their CEP, treatment goals, and decision factors considered when seeking out or selecting a treatment. This meeting is being conducted as a parallel effort to FDA’s PFDD initiative to more systematically gather patients’ perspectives on their conditions and available therapies to treat their conditions. United Porphyrias Association was honored to serve as the host organization and grateful for the FDA’s attendance.
CEP patients and family members joined us in-person and online from around the world, and brought to life the painful realities of living with CEP.
Thank you to everyone who participated, together we can change the world for CEP.
“I’ve prayed for this moment for 24 years, for my daughter to have others to speak with about her disorder and to have hope for a cure or help from the medical community.”
Meeting Outcomes
The UPA has produced a Voice of the Patient report summarizing the experiences and priorities identified during the meeting. This report has been submitted to the FDA and made publicly available to inform regulatory decisions and future drug development.
Other meeting documents:
Thank you to Disc Medicine for their regulatory support of the meeting.