PWSA | USA is excited to share an update on the continuous advocacy efforts for the PWS community. We have received a letter from the U.S. Food and Drug Administration (FDA) in response to a petition submitted by the PWS Advocacy Coalition (submitted on July 31, 2024). This petition urged the FDA to prioritize the approval of Soleno Therapeutics’ New Drug Application (NDA) for DCCR (diazoxide choline), a potential treatment for Prader-Willi syndrome (PWS). You can read the full petition here.
In their response letter, the FDA acknowledged the profound challenges that families affected by PWS face every day. They expressed appreciation for the collective efforts of the PWS community in raising awareness and contributing to the conversation surrounding DCCR. Importantly, the FDA underscored their commitment to supporting rare disease treatments and accelerating the development of therapies for conditions like PWS.
While the FDA cannot publicly disclose specific details about pending drug applications, the letter assures us they are working closely with drug developers to bring safe and effective treatments to market as swiftly as possible.
This response signals significant recognition of the PWS community’s advocacy work and strengthens our optimism for the future of DCCR. We remain hopeful and will continue to push for meaningful progress.
Stay tuned for more updates as we continue this critical dialogue with the FDA, and thank you for being a part of this journey to secure new treatment options for PWS.
Click the button below to read the letter from the FDA.
Jennifer Bolander has been serving as a Special Education Specialist for PWSA (USA) since October of 2015. She is a graduate of John Carroll University and lives in Ohio with her husband Brad and daughters Kate (17), and Sophia (13) who was born with PWS.
Perry A. Zirkel has written more than 1,500 publications on various aspects of school law, with an emphasis on legal issues in special education. He writes a regular column for NAESP’s Principal magazine and NASP’s Communiqué newsletter, and he did so previously for Phi Delta Kappan and Teaching Exceptional Children.
Evan has worked with the Prader-Willi Syndrome Association (USA) since 2007 primarily as a Crisis Intervention and Family Support Counselor. Evans works with parents and schools to foster strong collaborative relationships and appropriate educational environments for students with PWS.
Dr. Amy McTighe is the PWS Program Manager and Inpatient Teacher at the Center for Prader-Willi Syndrome at the Children’s Institute of Pittsburgh. She graduated from Duquesne University receiving her Bachelor’s and Master’s degree in Education with a focus on elementary education, special education, and language arts.
Staci Zimmerman works for Prader-Willi Syndrome Association of Colorado as an Individualized Education Program (IEP) consultant. Staci collaborates with the PWS multi-disciplinary clinic at the Children’s Hospital in Denver supporting families and school districts around the United States with their child’s Individual Educational Plan.
Founded in 2001, SDLC is a non-profit legal services organization dedicated to protecting and advancing the legal rights of people with disabilities throughout the South. It partners with the Southern Poverty Law Center, Protection and Advocacy (P&A) programs, Legal Services Corporations (LSC) and disability organizations on major, systemic disability rights issues involving the Individuals with Disabilities Education Act (IDEA), Americans with Disabilities Act (ADA), and the federal Medicaid Act. Recently in November 2014, Jim retired.