Patient-Focused Drug Development Meeting on MELAS
MitoAction is honored to host a Patient-Focused Drug Development (PFDD) meeting focused on MELAS.
Virtual PFDD Meeting Scheduled for February 10, 2026 – More Details Coming Soon
MitoAction is honored to host a Patient-Focused Drug Development (PFDD) meeting focused on MELAS (MitochondrialRelated to the mitochondria. Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes). The virtual meeting will be held on February 10, 2026, and is open to all members of the mitochondrial disease community, including patients, caregivers, clinicians, researchers, and industry partners.
“MELAS 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 MELAS—and those who support them—to attend and be part of shaping the future of mitochondrial disease care and treatment.
Patient-Focused Drug Development Meeting focused on MELAS
Date: February 10, 2026
Time: To Be Announced
Location: Virtual (Registration details to be announced)
For updates and registration information, please contact info@mitoaction.org.
Frequently Asked Questions
What is a Patient-Focused Drug Development (PFDD) meeting?
A PFDD is a public FDA meeting designed to engage patients and elicit their perspectives on two topic areas: (1) the most significant symptoms of their condition and the impact of the condition on daily life; and, (2) their current approaches to treatment.
PFDD meetings provide key stakeholders—including the FDA—patient advocates, researchers, drug developers, healthcare providers, and others, an opportunity to hear the patient’s voice. The lessons learned include experiences that matter most to patients, patient perspectives on meaningful treatment benefits and how patients want to be engaged in the drug development process.
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 published on their website. 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 Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS)?
Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS) is a rare genetic mitochondrial syndrome that can present in childhood or, more commonly, in adulthood. Along with the stroke-like episodes and lactic acidosis, other symptoms of MELAS include muscle weakness, fatigueThe overall feeling of tiredness or lack of energy. It is not the same as simply feeling drowsy or sleepy. Being fatigued means having no motivation or energy., hearing loss, and progressive neurological decline. The condition significantly affects daily functioning and quality of life, with no cure currently available. Treatment is limited to symptom management and supportive care. Learn more about MELAS.
