(Sept. 8, 2023) Earlier today, the United Mitochondrial Disease Foundation (UMDF) cohosted a listening session with the U.S. Food and Drug Administration (FDA) with fellow advocacy group MitoAction regarding the mitochondrial disease Pyruvate Dehydrogenase Complex Deficiency (PDCD).
The closed session, “The Voice of Pyruvate Dehydrogenase Complex Deficiency Patient Community”, was requested by members of the PDCD community to help the FDA understand the burden of the disease and the need for an approved therapy.
“I think the message from PDCD patients and caregivers to the FDA today was very clear – this disease needs an approved treatment. Families are doing everything they can with the resources at their disposal, but PDCD is a devastatingly progressive disease. This is a community eager for clinical trials,” said UMDF President and Chief Executive Officer Brian Harman. “It’s critical that the voices of expertise – patients, families, clinicians, and researchers – continue to be heard as we work together toward making an approved PDCD treatment a reality.”
Six caregivers – including one caregiver who was also an affected adult – to PDCD patients participated in the FDA videoconference, along with clinician Rebecca Ganetzky, MD, of Children’s Hospital of Philadelphia.
Said Emma, whose daughter Elizabeth is a 20-year-old patient with PDCD: “As a family we have lived in a crisis mode for the past 20 years, constantly worried about death. We have already watched her regress medically and physically and we fear that every illness will be the one that ends her life. Even minor illnesses can be catastrophic, so we live all day every day in fear.”
UMDF thanks the PDCD community members who shared their stories, the FDA team members who intently listened, and the many patient advocacy groups that helped make the listening session a success including MitoAction, Hope for PDCD, and The Elizabeth Watt PDCD Research Fund.
This was the second FDA Listening Session UMDF has cohosted for a mitochondrial disease in the last two years. In January 2022, the FDA held a similar session for thymidine kinase 2 deficiency (TK2d), another rare mitochondrial disease.
A full report of the FDA Listening Session for PDCD is expected in early October. Stay tuned to UMDF social media for more information.