On Thursday, August 4, 2022, six members of the SMA community had 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 was a 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 was 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, progress in the SMA treatment landscape has been profound and rapid, resulting in FDA approval of three SMN-upregulating therapeutics. These treatments, together with the fact that 97% of newborns in the U.S. are now screened for SMA, have resulted in a quickly evolving disease outcomes. However, significant unmet needs remain in those who are treated later in life, and other needs will arise in individuals who were treated early in life as they age. The goal of this session was to provide a forum in which six SMA patient advocates, each with unique disease and treatment experiences, could tell their stories and describe their unmet medical needs.
This 90-minute session began with opening remarks by the FDA and Cure SMA. The next 30 minutes of the session were dedicated to testimony from six SMA-affected individuals or their caregivers. Each of the advocates discussed the need for additional therapeutics and further research.
Testimonies from the FDA Listening Session
“Today, our biggest concern is what the future holds for Declan. Will we be able to tell if symptoms begin as we know early intervention is best? And will there be effective treatments for these symptoms when they occur? The development of add-on, non-SMN targeted therapies so that there are additional options to treat future needs as they arise would provide our family a great deal of peace of mind about what the future holds for Declan and our family.” – Maria Demeke, mom to Declan, age 16 months, treated presymptomatically.
“I turned 16 this year, unlike most of my friends, I did not get my driver’s license. Even though my muscles are stronger than in the past, I get tired really easily and don’t feel like driving a car would be safe with my neck control issues. For me a treatment that targets muscle gain would greatly improve my independence and would ensure that i can continue doing the things that i love like power soccer.” – Wren Grabham, age 16
“Small tasks are huge success in my life. If I could lift that 1L bottle of water at work instead of having to find a graduate student to move it for me or lift a half gallon of milk in and out of the fridge to cook with instead of calling for someone to come help. If I could reach my arms up to put my hair in a ponytail. These things don’t take a ton more muscle, but they are all muscle I still don’t have. If science can create a safe effective treatment that helps me maintain, I fully believe and expect it will be able to create a combination treatment to allow me to gain some strength.” – Sarah Boggess, adult with SMA
Following the testimonies of each of the advocates, representatives from the FDA were able to ask questions. Among topics addressed were combination/add-on therapies and needs the specific needs advocates would like to see addressed by these types of therapies, barriers to clinical trial participation, and gratitude for existing treatment options.
To advocates: Amy Medina, Maria Demeke, Kelly Eakin, Wren Grabham, Kyle Derkowski, and Sarah Boggess for their participation and willingness to share their lived experience with SMA.