MitoAction, POLG Foundation set virtual drug development meeting on rare mitochondrial disorders

MitoAction and The POLG Foundation will host a virtual patient-focused drug development meeting Nov. 17, 2026, to give people affected by POLG-related disorders a direct channel to the FDA and other stakeholders. The event aims to capture lived experience, inform future research and treatment development, and feed into an official Voice of the Patient report.

Why it matters: - POLG-related disorders are rare, progressive mitochondrial diseases with no cure and limited treatment options beyond symptom management. - The Nov. 17 meeting gives patients and caregivers a formal platform to shape how the FDA and other stakeholders understand the real-world burden of the disease. - The testimony gathered will help inform future research, therapy development and regulatory decisions.

What happened: - MitoAction and The POLG Foundation announced they will co-host an Externally-Led Patient-Focused Drug Development meeting on POLG-related disorders. - The virtual event is scheduled for Tuesday, Nov. 17, 2026, from 10:00 AM to 4:00 PM ET. - The meeting is open to patients, caregivers, clinicians, researchers and industry partners across the mitochondrial disease community. - The event will be produced at Detroit Country Day School's state-of-the-art film and virtual production studio.

The details: - Participants will be able to share experiences through live testimony, polling and written comments. - Patients and caregivers can complete a 30-minute anonymous pre-meeting survey through Oct. 31, 2026. - Personal stories submitted ahead of the meeting will be compiled into the official Voice of the Patient report shared with the FDA. - Registration is available here. - The pre-meeting survey is available here. - Updates and registration information are available at more information. - MitoAction says the meeting is intended to reflect the full range of POLG experiences, from children with Alpers-Huttenlocher syndrome to adults living with ataxia and PEO. - Advocacy partners including the United Mitochondrial Disease Foundation and International Mito Patients are helping bring the meeting forward. - POLG-related disorders are caused by changes in the POLG gene, which provides instructions for making polymerase gamma, the only enzyme that can copy and repair mitochondrial DNA. - The POLG spectrum includes Alpers-Huttenlocher syndrome, ataxia neuropathy spectrum, childhood myocerebrohepatopathy spectrum, myoclonic epilepsy myopathy sensory ataxia, progressive external ophthalmoplegia and others. - These disorders most often affect high-energy organs such as the brain and muscles, and symptoms can vary widely.

Between the lines: - Patient-focused drug development meetings can influence how regulators and drug developers weigh symptoms, functional impact and meaningful treatment benefit. - The event's focus on live testimony and a Voice of the Patient report signals an effort to elevate patient experience alongside clinical data. - The explicit push for survey responses suggests organizers want a broad set of firsthand accounts before the meeting begins.

What's next: - Patients and caregivers can submit survey responses and stories through Oct. 31, 2026. - The community will gather virtually on Nov. 17, 2026, for the formal PFDD session. - The compiled patient input will be shared with the FDA after the meeting.

The bottom line: - MitoAction and The POLG Foundation are using a rare-disease patient forum to translate lived experience into input that could shape future POLG research, treatment development and regulatory review.