Soleno Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has accepted their New Drug Application (NDA) and granted priority review for DCCR (Diazoxide Choline Controlled-Release), a treatment for patients with Prader-Willi syndrome. The FDA's target action date (the goal date to have completed the application review) is December 27, 2024. This timeline is not guaranteed and the review may extend past this date if additional information or analysis is required.
In its Priority Review Designation letter, the FDA stated its intent to hold an advisory committee meeting to discuss the application for DCCR. This meeting will involve a panel of independent experts, including physicians, scientists, and patient representatives, who will assess the data provided by Soleno Therapeutics. The committee will discuss the drug’s potential benefits and risks, hear testimonies, and consider public input. After a thorough discussion, the panel will vote on critical issues, including whether the FDA should approve the drug. Although the FDA is not obligated to follow the committee’s recommendations, these votes are highly influential in the final decision. FPWR and PWSA-USA will conduct an informational webinar (date to be announced) to share more on advisory committees and how the PWS community can be involved in the process.
The FDA's acceptance of the application marks a critical milestone as DCCR moves closer to potential approval. Stay tuned for further updates as Soleno continues to navigate the FDA's review process.
You can read Soleno's full press release below.
REDWOOD CITY, Calif., Aug. 27, 2024 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing its New Drug Application (NDA) for DCCR for the treatment of Prader-Willi syndrome (PWS) in individuals four years and older who have hyperphagia. The FDA granted Priority Review for the NDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of December 27, 2024. Priority review is designated to applications for drugs that, if approved, would provide a significant improvement in safety or effectiveness of the treatment, prevention or diagnosis of a serious condition.
In its Priority Review Designation letter, the FDA also stated that it is currently planning to hold an advisory committee meeting to discuss the application for DCCR.
“PWS is a devastating, rare condition that significantly impacts the lives of patients and their families,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno. “The FDA’s acceptance of our NDA is a significant milestone, and designating our application for priority review reaffirms that the FDA views PWS as a serious condition. We are immensely grateful to the entire PWS community, including patients, caregivers and advocacy groups, for their ongoing support. We remain committed to continuing to work closely with the FDA through the NDA review process.”
Diazoxide choline has Breakthrough and Fast Track Designations in the U.S., as well as Orphan Drug Designation for PWS in the U.S. and E.U.
About PWS
The Prader-Willi Syndrome Association USA estimates that PWS occurs in one in every 15,000 live births. The hallmark symptom of this disorder is hyperphagia, a chronic and life-threatening condition characterized by feelings of intense, persistent hunger, food pre-occupation, and an extreme drive to seek and consume food, which can severely diminish the quality of life for individuals with PWS and their families. Additional characteristics of PWS include behavioral problems, cognitive disabilities, low muscle tone, short stature (when not treated with growth hormone), the accumulation of excess body fat, developmental delays, and incomplete sexual development. Hyperphagia can lead to significant mortality (e.g., stomach rupture, choking, accidental death due to food seeking behavior) and longer term, co-morbidities such as diabetes, obesity, and cardiovascular disease. In a global survey conducted by the Foundation for Prader-Willi Research, 96.5% of respondents (parents and caregivers) rated hyperphagia and 92.9% rated body composition as either the most important or a very important symptom to be relieved by a new medicine. There are currently no approved therapies to treat the hyperphagia/appetite, metabolic, cognitive function, or behavioral aspects of the disorder.
About DCCR (Diazoxide Choline) Extended-Release Tablets
DCCR is a novel, proprietary extended-release dosage form containing diazoxide choline, the crystalline salt of diazoxide and is administered once-daily. The parent molecule, diazoxide, has been used for decades in thousands of individuals in a few rare diseases in neonates, infants, children and adults, but is not approved for use in PWS. Soleno conceived of and established extensive patent protection for the therapeutic use of diazoxide, diazoxide choline and DCCR in individuals with PWS. The DCCR development program is supported by data from five completed Phase 1 clinical studies in healthy volunteers and three completed Phase 2 clinical studies, one of which was in individuals with PWS. In the PWS Phase 3 clinical development program, DCCR showed promise in addressing hyperphagia, the hallmark symptom of PWS, as well as several other symptoms such as aggressive/destructive behaviors, fat mass and other metabolic parameters.
About Soleno Therapeutics, Inc.
Soleno is focused on the development and commercialization of novel therapeutics for the treatment of rare diseases. An NDA for its lead candidate, DCCR (diazoxide choline) extended-release tablets, a once-daily oral tablet for the treatment of Prader-Willi syndrome (PWS) is currently under review by the FDA and was granted Priority Review. For more information, please visit www.soleno.life.
