HDSA to Host Externally-led Patient Focused Drug Development (PFDD) Meeting

FDA Recognizes Value in Additional PFDD Meeting for Huntington’s Disease

New York, NY (April 16, 2024) — The Huntington’s Disease Society of America (HDSA) has received approval from the U.S. Food and Drug Administration (FDA) to host an Externally-led Patient Focused Drug Development (EL-PFDD) meeting this year.  Currently planned for late fall 2024, this important meeting will focus on the needs of people in stages 1 and 2 of Huntington’s disease (HD), which is commonly known as the pre-symptomatic and earlier symptomatic stages of the disease. 

The FDA previously met with Huntington’s disease patients and families in 2015 when the patient focused drug-development meeting program was in its infancy. With HDSA’s approval, the FDA has recognized the value of this externally-led PFDD meeting, which will provide a platform for the patient community, patient advocates, researchers, drug developers, and the FDA to come together.

“A lot has changed in HD clinical science since the FDA’s first meeting with the community nearly a decade ago,” said Louise Vetter, President & CEO of the Huntington’s Disease Society of America.  “Today the HD community is focused on the development of disease-modifying therapies that can slow and potentially stop the trajectory of this fatal, genetic illness.  As research and clinical trials are focusing on people in earlier stages, we are thrilled that FDA has recognized the importance of an additional meeting to learn from these families.”

HDSA’s commitment to improving the lives of those affected by HD and their families is at the forefront of this initiative. By raising awareness of the urgent need to accelerate the development of new treatments, HDSA aims to support the FDA and medical product developers in capturing the needs of patients and families when developing new therapies and making regulatory decisions. The organization seeks to amplify the voices of HD families, addressing the burden of living with and supporting someone with HD, preferred treatment outcomes and wellness priorities, challenges of participating in clinical trials, and benefit expectations and risk tolerance for new therapies.

This hybrid PFDD meeting will provide HD families with a unique opportunity to share their experiences directly with the FDA and medical product developers. The meeting will feature six patient and family member panelists who will share their personal stories, shedding light on the challenges and triumphs of living with HD. Additionally, the meeting will offer a forum for HD families to share their own experiences, with virtual attendees having the chance to participate in polling questions and share their perspectives.

“We are pleased that the FDA has acknowledged the need for a second PFDD meeting on Huntington’s disease. This demonstrates the value in gathering additional information from the HD community based on the advances in research to treat HD,” stated Arik Johnson, PsyD, HDSA’s Chief Mission Officer.

The input gathered during this meeting will play a crucial role in informing the FDA’s decisions and oversight throughout the drug development process and the review of new applications for HD therapies. This is an opportunity to make a difference and contribute to the advancement of HD treatments.

HDSA encourages all interested parties, including the HDSA Community, the broader HD community, sister HD organizations, and industry partners, to keep an eye out for future updates regarding the date and location of the PFDD meeting.

For more information and updates, please visit the HDSA website at www.hdsa.org.


Huntington’s disease (HD) is a fatal genetic disorder that causes the progressive breakdown of nerve cells in the brain. It deteriorates a person’s physical and mental abilities during their prime working years and has no cure. Every child of a parent with HD has a 50/50 chance of inheriting the faulty gene that causes HD. Today, there are approximately 41,000 symptomatic Americans and more than 200,000 at risk of inheriting the disease. The symptoms of HD are described as having, ALS, Parkinson’s and Alzheimer’s – simultaneously.

The Huntington’s Disease Society of America is the premier nonprofit organization dedicated to improving the lives of everyone affected by Huntington’s disease. HDSA supports research to find a cure for HD, provides resources and support services to HD families, educates the public and healthcare professionals about HD, and advocates for legislation and policies that benefit the HD community.



Mynelly Perez
Director of Marketing & Communications
212-242-1968, ext. 214