Soleno Therapeutics Submits FDA Application for DCCR Tablets to Treat Prader-Willi Syndrome

On June 28, 2024, Soleno Therapeutics, Inc. (NASDAQ: SLNO), a biopharmaceutical company focused on developing treatments for rare diseases, announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA). The application seeks approval for DCCR (diazoxide choline) extended-release tablets for treating Prader-Willi syndrome (PWS) in individuals aged four years and older who suffer from hyperphagia.

Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics, emphasized the importance of this milestone. He noted that the regulatory submission represents a significant step toward providing a new therapeutic option for individuals with PWS, a condition characterized by life-threatening hyperphagia and other severe symptoms. Bhatnagar expressed gratitude to the Soleno team, investigators, study sites, advocacy groups, and especially the families affected by PWS, who played a crucial role in completing the DCCR development program.

DCCR has already received Breakthrough and Fast Track Designations in the U.S. Additionally, it has been granted Orphan Drug Designation for PWS in both the U.S. and the European Union. The FDA will have 60 days to decide whether to accept the NDA for review. Soleno has also requested Priority Review, which would set a target review period of six months post-acceptance.

Prader-Willi syndrome is estimated to occur in one in every 15,000 live births, according to the Prader-Willi Syndrome Association USA. The disorder's primary symptom, hyperphagia, involves a chronic and intense hunger, a preoccupation with food, and a compulsion to seek and consume food. These symptoms can severely impact the quality of life for individuals with PWS and their families. Other characteristics of the syndrome include behavioral issues, cognitive disabilities, low muscle tone, short stature (when untreated with growth hormone), excessive body fat, developmental delays, and incomplete sexual development. Hyperphagia can lead to severe health risks, including stomach rupture, choking, and accidental death from food-seeking behaviors, as well as long-term conditions like diabetes, obesity, and cardiovascular disease.

A global survey by the Foundation for Prader-Willi Research highlighted the urgency for effective treatments: 96.5% of respondents, comprising parents and caregivers, rated hyperphagia as a critical symptom needing relief, while 92.9% highlighted body composition as a significant concern. Currently, there are no approved therapies to address the hyperphagia, metabolic, cognitive, or behavioral aspects of PWS.

DCCR, or diazoxide choline, is a proprietary extended-release formulation administered once daily. The parent compound, diazoxide, has been used for decades in treating various rare diseases in neonates, infants, children, and adults, although it is not currently approved for PWS. Soleno has secured extensive patent protection for the use of diazoxide, diazoxide choline, and DCCR in treating PWS. The DCCR development program is backed by data from five Phase 1 clinical studies in healthy volunteers and three Phase 2 studies, including one specific to PWS. In Phase 3 clinical trials for PWS, DCCR demonstrated potential in managing hyperphagia and other symptoms such as aggressive/destructive behaviors, fat mass, and metabolic parameters.

In conclusion, Soleno Therapeutics is making strides in the development and commercialization of novel treatments for rare diseases. The submission of the NDA for DCCR extended-release tablets is a significant development in the quest to offer a new therapeutic option for individuals affected by Prader-Willi syndrome.