Cure SMA to Hold Patient-Led Listening Session with FDA

On Thursday, August 4, 2022, six members of the SMA community will have the opportunity to talk directly with the U.S. Food and Drug Administration (FDA) about their lived experiences with SMA and their unmet medical needs. This Patient-Led Listening Session will be a small, private teleconference attended by patient advocates, FDA representatives from the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER), and representatives from Cure SMA.

The purpose of this listening session is to give members of the SMA community a forum in which they can offer their perspectives on current and future SMA therapeutics. In recent years, incredible progress in SMA research and development has culminated in FDA approval of three SMN-upregulating therapeutics. These therapeutics, along with the implementation of newborn screening and improvements in diagnosis and care, have been life-changing for many individuals living with SMA. However, significant medical needs remain for SMA-affected individuals who were not treated pre-symptomatically, and new needs have arisen in treated individuals. Additional therapeutic strategies, such as combination therapies, are necessary to address these needs.

This 90-minute session will begin with opening remarks by the FDA and Cure SMA. The next 30 minutes of the session will be dedicated to testimony from six SMA-affected individuals or their caregivers. These representatives of the SMA community will include the parents of children who were treated both presymptomatically and symptomatically as well as a teen and two adults who were treated symptomatically. Each person will have the opportunity to describe their unique lived experiences with SMA and offer their perspectives on critical unmet needs for the SMA community as well as what they would like to see in new therapeutics. Following these advocate statements, there will be a Q and A session for FDA to ask questions of the advocates and representatives.

Although remarkable breakthroughs in SMA therapeutics have been made in recent years, it is critical to continue research and drug development to address the remaining unmet needs experienced by the SMA community. This patient listening session is an important opportunity for members of the SMA community to meet face-to-face with the FDA and communicate their perspectives on these needs. Following the listening session, a summary will be posted by the FDA and on the Cure SMA website.

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