On October 20, the US Food and Drug Administration (FDA) will host a Patient Listening Session for the CMT2C/TRPV4 community, organized by CMTA. During this 1.5-hour virtual event, FDA decision-makers will hear from individuals and families living with this type of CMT about their lives, their needs, and their hopes for new treatments.
What is an FDA Patient Listening Session?
A Patient Listening Session is a private, informal meeting between the FDA and a patient group, such as the Charcot-Marie-Tooth community. During these sessions, the FDA hears directly from individuals, caregivers, and medical experts about life with that rare disease. The goal is to listen and learn.
Listening sessions are not tied to a single drug trial or application. They allow the FDA to ask questions and learn from people who live with the disease. The FDA then summarizes these sessions, which are used to inform regulatory decisions and educate the public.
In rare disease, patient input carries more weight
CMT2C (also called TRPV4-associated CMT) is a rare and severe form of Charcot-Marie-Tooth caused by changes in the TRPV4 gene. It affects about 2,500 people in the United States. Along with muscle weakness, many CMT2C patients experience vocal cord paralysis, breathing problems, and labored speech — symptoms that worsen over time.
Because CMT2C is so rare, little is known about it. With limited research and small clinical trials, the FDA has less evidence to make a decision than usual. The FDA staff reviewing a treatment for CMT2C may not understand the disease as well as they understand common conditions like diabetes or asthma.
This is why patient input is critical. A Patient Listening Session may be the first time the FDA hears firsthand what a disease is really like from those living with it. Listening sessions help the FDA understand which symptoms are most bothersome, what meaningful physical improvement means, and how the disease changes over time. This input shapes how the FDA considers a new treatment for approval.
Why a CMT2C/TRVP4 Listening Session?
Research on CMT2C/TRVP4 is advancing, with potential treatments moving out of the laboratory and toward testing in people. For a disease with no approved therapy, that progress makes the patient perspective especially timely. What the FDA hears now can help shape the research to come.
At the 90-minute CMT2C/TRVP4 session, the FDA will hear directly from people with CMT2C — voices spanning generations, from a young child to adults who have lived with the disease for years. They’ll speak to what the disease takes from everyday life, how it changes over time, and what a treatment would mean for them and the people they love.
The CMTA organized the October 20 Listening Session with the FDA on behalf of the CMT2C/TRPV4 community. Giving people with CMT a direct voice in research is one way we speed treatments toward every type of CMT.
If you want to use your voice and experience to shape CMT research, join CMTA’s Patients as Partners in Research to receive the latest opportunities.
The information provided here is for informational purposes only and does not constitute medical advice. The CMTA does not endorse any specific products or treatments for CMT. Please consult your doctor before making any healthcare decisions.
