Externally Led Patient-Focused Drug Development Meeting on PCQD
MitoAction is honored to host a Patient-Focused Drug Development (PFDD) meeting focused on PCQD.
MitoAction is honored to host an Externally Led Patient-Focused Drug Development (PFDD) meeting focused on Primary CoQ10 Deficiency (PCQD). This meeting is open to all members of the mitochondrial disease community, including patients, caregivers, clinicians, researchers, and industry partners.
“PCQD deeply impacts every aspect of life for patients and families. This PFDD meeting is a crucial platform for our community to educate regulators, researchers, and developers about the urgent needs and challenges of living with this devastating condition.
Kira Mann, CEO of MitoAction
Participants will be invited to share their experiences through live testimony, polling, and written comments. MitoAction encourages all individuals and families affected by Primary CoQ10 Deficiency—and those who support them—to attend and be part of shaping the future of mitochondrial disease care and treatment.
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Externally Led Patient-Focused Drug Development Meeting focused on PCQD
Date: Tuesday, February 16, 2027
Time: Coming Soon
Location: Coming Soon
For updates and registration information, please contact info@mitoaction.org.
Frequently Asked Questions
What is an Externally Led Patient-Focused Drug Development (PFDD) meeting?
Externally-led Patient-Focused Drug Development meetings give the FDA and other key stakeholders—including medical product developers, health care providers, and federal partners—an important opportunity to hear directly from patients, their families, caregivers, and patient advocates about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, and patients’ experiences with currently available treatments. This input can inform the FDA’s decisions and oversight both during drug development and during review of a marketing application.
Why should I participate in a PFDD meeting?
This PFDD meeting is a critical opportunity to elevate the voice of those impacted by MELAS, helping the FDA and other key stakeholders understand the real-life burden of this complex and progressive mitochondrial disorder. Insights shared during the meeting will help guide future research, therapy development, and regulatory decisions. MitoAction will be working alongside advocacy partners including UMDF, IMP, and MDA among others to bring this meeting forward.
What is the outcome from PFDD meetings?
Following the PFDD meeting, MitoAction will summarize the input shared by patients and patient representatives in a Voice of the Patient report, which will be shared with the FDA and other key stakeholders. Clinicians, researchers, and industry partners will continue to refer to the summary report as they work on new therapies and treatments. Links to meeting materials, including transcripts, webcast recordings, presentation slides, and Voice of the Patient reports can be found on the Condition-Specific Meeting Reports webpage.
What is Primary CoQ10 Deficiency (PCQD)?
Coenzyme Q10 is a lipid in the ETC that helps move electrons from complexes 1 and 2 to complex 3 in the ETC. It also receives electrons from other proteins in mitochondria supporting mitochondrial function. Primary CoQ10 deficiency happens when there is a genetic mutation that doesn’t allow the body to make enough CoQ10 that’s needed by the mitochondria to function normally.
