Media
Media Inquiries
We welcome media interest in this effort and in the stories of people living with EPP and XLP.
Patients for Bitopertin can help connect members of the media with patients, caregivers, and families willing to share their experiences living with these painful, life-limiting rare diseases, as well as the impact bitopertin has had for those who have received it.
If you are a member of the media and are interested in covering this story, featuring a patient voice, or learning more about the urgency facing this community, please contact us.
In the Media
Explore recent media coverage, interviews, and stories related to EPP, XLP, bitopertin, and the FDA announcements regarding rare disease therapies.
Porphyria News: A patient-voice commentary piece reflecting on the FDA decision and why the community remains hopeful while facing the emotional reality of the delay.
Boston 25 News: Ginger Gonsalves and Becky Kelliher joined Kerry Kavanaugh to discuss their experience living with EPP and their positive experience on bitopertin, and to advocate for approval of bitopertin.
FDA Press Announcement: The FDA announced a new framework and draft guidance aimed at helping sponsors generate evidence of safety and effectiveness when randomized trials are not feasible because patient populations are extremely small. This is one of the clearest recent statements of FDA intent to create more workable pathways for ultra-rare disease therapies.
BioWorld: A piece focused on the regulatory angle, including questions about consistency and stability at the FDA after a CRL was issued for a drug that had been designated under the Commissioner’s National Priority Voucher pathway.
Fierce Biotech: This story zeroes in on the pathway issue, explaining that the FDA agreed the trial was adequate and well-controlled but objected to the surrogate endpoint and the lack of a sufficiently demonstrated link to clinical outcomes.
Reuters: A clear overview of the CRL, including that bitopertin had been reviewed under the FDA’s Commissioner’s National Priority Voucher program and that the agency did not find enough evidence linking the biomarker response to clinical benefit.
People: Craig Leppert shares his experience living with EPP, founding Shadow Jumpers, and what bitopertin has meant for people with this painful rare disease. The story helps bring the human side of EPP and treatment access into clear view.
FDA Rare Disease Innovation Hub: The FDA Rare Disease Innovation Hub released its Strategic Agenda, outlining priorities for the year and emphasizing partnership with the rare disease community, regulatory innovation, and improved development pathways for rare disease products.
The Boston Globe: A strong human-interest story that helps explain what bitopertin can mean in real life for people with EPP.
ABC 10News San Diego: This story features Sophia McClellan, a University of San Diego student from Southern California living with EPP. It focuses on how EPP shaped her life and academic path
FDA Press Announcement: The FDA introduced the Rare Disease Evidence Principles to provide greater speed and predictability for therapies intended for very small rare disease populations with significant unmet need. The announcement specifically highlights clearer guidance on the kinds of evidence that may support approval in these settings.