In an important milestone for the CEP community and rare disease advocacy, United Porphyrias Association has received official acceptance from the U.S. Food and Drug Administration (FDA) to host an Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting on Congenital Erythropoietic Porphyria (CEP)!

This meeting will bring members of the CEP community, advocates, porphyria experts and the FDA together to share experiences, influence research priorities, and advocate for better treatments.



What to Expect

The meeting include:

• Two CEP Patient Panels. This gives the opportunity to share the patient voice regarding burden of disease and unmet therapeutic need to the agency that regulates therapies.

• Discussions focused on daily impact, treatment experiences, and unmet needs

• A final Voice of the Patient report submitted to the FDA and made publicly available to inform regulatory decisions and future drug development

We are honored to serve as the host organization and grateful for the FDA’s partnership and support.

Are you a CEP patient or caregiver for someone with CEP?

We’d love to discuss how you can participate. Please contact us via the form below.