Soleno Therapeutics continues to progress their novel therapeutic, DCCR, forward as a potential treatment of hyperphagia in PWS. On November 12th, Soleno met with the FDA to discuss the best path forward for DCCR. In their most recent press release, they summarize their work to date.
Soleno presented data from their Phase 3 Study- DESTINY-PWS in October at FPWR's Annual Family Conference. The recorded session can be viewed on FPWR's YouTube Channel.
Dec. 11, 2020. Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced the receipt of official meeting minutes from the November 12, 2020, Type C meeting with the U.S. Food and Drug Administration (FDA) regarding the development of once-daily Diazoxide Choline Controlled Release (DCCR) tablets for the treatment of Prader-Willi Syndrome (PWS). The meeting minutes confirmed the discussion with the FDA regarding the potential adequacy of data from completed and ongoing studies with DCCR, together with external, natural history studies to support a New Drug Application (NDA) for DCCR for the treatment of PWS. Soleno intends to submit formal plans for these analyses, which the FDA has stated it is committed to reviewing.
“We appreciate the guidance provided by the FDA and are finalizing our plans to conduct the analyses that were discussed at this meeting and look forward to submitting them to the FDA,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics. “While we hope that these data, together with the additional analyses, will suffice as adequate for an NDA, we cannot, at this time, exclude the possibility that the FDA will require us to conduct an additional controlled clinical study. We look forward to working with the FDA to ensure that the necessary data and information are compiled for the NDA, which we are currently preparing to submit in the second half of 2021.”
The Prader-Willi Syndrome Association USA estimates that PWS occurs in one in every 15,000 live births in the U.S. In a global survey conducted by the Foundation for Prader-Willi Research, 96.5% of respondents (parent and caregivers) rated hyperphagia as 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. Diazoxide choline has received Orphan Drug Designation for the treatment of PWS in the U.S. and EU, and Fast Track Designation in the U.S.
About Diazoxide Choline Controlled-Release (DCCR) Tablet
Diazoxide Choline Controlled-Release tablet is a novel, proprietary extended-release, crystalline salt formulation of diazoxide, which is administered once-daily. The parent molecule, diazoxide, has been used for decades in thousands of patients in a few rare diseases in neonates, infants, children and adults, but has not been approved for use in PWS. Soleno conceived of and established extensive patent protection on the therapeutic use of diazoxide and DCCR in patients with PWS. The DCCR development program is supported by data from five completed Phase I clinical studies in healthy volunteers and three completed Phase II clinical studies, one of which was in PWS patients. In the PWS Phase III study, 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. The company’s lead candidate, Diazoxide Choline Controlled-Release (DCCR) tablets, a once-daily oral tablet for the treatment of Prader-Willi Syndrome (PWS), is currently being evaluated in a Phase III clinical development program. For more information, please visit www.soleno.life.