We are thrilled to share with you this BREAKING NEWS for the PWS Community! The U.S. Food and Drug Administration (FDA) has officially approved VYKAT™ XR (diazoxide choline extended-release tablets, formerly referred to as DCCR) as the first-ever treatment for hyperphagia in Prader-Willi syndrome. This is a monumental victory for individuals with PWS, our families, and the entire rare disease community. This approval is a testament to the unwavering dedication of advocates, researchers, and families who never gave up hope. Today, we celebrate a future with greater possibilities, more hope, and meaningful change for those living with PWS!
We also extend our deepest gratitude to Soleno Therapeutics for their commitment to the PWS community and for working hard to make this much-needed treatment a reality for our families and loved ones. To give further details, Soleno will host a Caregiver Now Approved Webinar on April 16th at 12:15 PM ET. Click here to register for this live event, which will be recorded.
“PWSA | USA and our incredible volunteer advocates played a critical role in achieving this historic milestone. For years, we have been the driving force in PWS advocacy – leading the charge through our D.C. Fly-In events, direct engagement with elected officials and FDA / Center for Drug Evaluation and Research (CDER) representatives, and the powerful testimonials shared at the 2023 Externally-Led Patient-Focused Drug Development meeting and beyond.
Our continuous, boots-on-the-ground efforts ensured that decision-makers heard our voices and understood the urgent need for treatment. This approval is not just a medical breakthrough, it’s the result of tireless dedication, strategic advocacy, and the unwavering belief that our loved ones deserve help and hope. I want to extend my deepest gratitude to our team and every single volunteer who helped make this moment possible. This is your victory.”
– Stacy Ward, MS, BCBA, PWSA | USA CEO
To learn more about this groundbreaking approval, we encourage you to:
- *Visit Soleno Therapeutics’ official VYKAT™ XR website where you’ll find additional details about the newly approved treatment.
- *Soleno and Medlive will host a Caregiver Now Approved Webinar on April 16th at 12:15 PM ET, in partnership with PWSA | USA and FPWR, where they will provide more in-depth information. You can register for this event by clicking here. This webinar will be recorded.
- *Additionally, we invite you to read Soleno’s official press release (Soleno Therapeutics Announces U.S. FDA Approval of VYKATTM XR to Treat Hyperphagia in Prader-Willi Syndrome – Soleno Therapeutics Inc.) for further details about this historic announcement.
We understand there will be many questions, and we kindly ask the PWS community to refer to Soleno’s website and press release for initial information and then join the April 16th webinar for further discussion. Please note that PWSA | USA staff does not have additional details beyond these resources. Thank you for your patience, and let’s continue celebrating this incredible milestone together!
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.
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.
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.
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.
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.