Externally Led Patient-Focused Drug Development Meeting on MELAS

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.

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.

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.

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.