On October 8, 2024, Soleno Therapeutics provided a regulatory update on DCCR (Diazoxide Choline Controlled-Release) tablets, which are currently undergoing priority review by the FDA as a potential treatment for hyperphagia in individuals with Prader-Willi syndrome (PWS). Although the FDA has moved forward with priority review, it has chosen not to hold an advisory committee meeting at this time.
We have worked diligently over the past several years to ensure that the patient voice is actively heard by FDA. Through initiatives like our patient listening session, the Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting, multiple engagements with the FDA, robust congressional support, and a community petition representing nearly 15,000 individuals, we continually strive to amplify the voices of those affected by PWS and advocate for access to effective treatments.
While it appears there may not be another formal opportunity for our community to share their support for DCCR prior to the Prescription Drug User Fee Act (PDUFA) date of December 27, 2024, we encourage you and/or your loved ones affected by PWS to contribute a written testimonial. PWSA | USA will gather and securely hold these testimonials, ensuring we are prepared should the FDA request additional community input. Testimonial submission is entirely optional, but we want to offer our community members another outlet to be heard in case additional input is needed.
If you or someone you love living with PWS would like to submit additional testimony, please fill out the form found by clicking the button below. We respectfully request that all submissions be received by November 3, 2024. Thank you for your ongoing support and advocacy. Together, we can continue to make a difference in bringing effective treatments to those living 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.
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.