On Tuesday, November 26, 2024, Soleno Therapeutics shared an important development regarding the New Drug Application (NDA) for DCCR (diazoxide choline) extended-release tablets—a potential treatment for individuals with Prader-Willi syndrome (PWS) aged four and older who experience hyperphagia.
The U.S. Food and Drug Administration (FDA) has extended the review period for this NDA, pushing the Prescription Drug User Fee Act (PDUFA) target action date to March 27, 2025 (originally December 27, 2024). Soleno’s press release states, “The FDA determined that responses to recent information requests constituted a major amendment to the NDA, resulting in the extension of the PDUFA goal date by three months. The extension allows the FDA time to complete their review, including that of the recently submitted information. The FDA did not cite any safety, efficacy, or manufacturing concerns in their correspondence.”
Why Was the Review Period Extended?
The FDA routinely reviews large volumes of data when evaluating a new drug application. If additional information is submitted during this process it can impact the review and the agency may extend its timeline to ensure thorough consideration. In this case, the three-month extension provides the FDA with the time it needs to review the recently provided details.
The Journey So Far
Soleno submitted its NDA for DCCR in June 2024, and the FDA granted Priority Review in August. Priority Review is reserved for drugs that could provide significant improvements in the treatment of serious conditions, underscoring the potential importance of DCCR for the PWS community. In addition, DCCR has received Breakthrough Therapy, Fast Track, and Orphan Drug designations in recognition of its potential to address critical unmet needs.
What Happens Next?
The new action date of March 27, 2025, means the FDA aims to complete its review and make a decision on the DCCR NDA at that time. Until then, Soleno says it will continue to work closely with the FDA to address any remaining questions.
“The FDA did not cite any safety, efficacy, or manufacturing concerns in their correspondence.”
While this extension may feel like a delay, it reflects the FDA’s commitment to ensuring all submitted information is thoroughly reviewed before making a decision. The good news is that no new concerns have been raised about DCCR’s safety or effectiveness.
We understand that the PWS community is eager for new treatment options, and we remain hopeful this extension will bring us one step closer to a positive outcome.
Stay Informed
PWSA | USA is committed to keeping the community updated throughout this process. We will share any new information as soon as it becomes available.
Thank you for your continued patience, support, and advocacy as we work together to advance treatments for PWS.
You can also sign up to receive updates from Soleno Therapeutics: https://support4pws.com/
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.