Soleno Therapeutics announced today that the FDA will require an additional controlled clinical trial to support an NDA submission for DCCR in PWS. This is despite pre-Covid data that showed statistically significant changes for DCCR compared to placebo in the primary and key secondary endpoints. “We intend to continue the dialogue with the FDA to ensure that DCCR is approved for individuals with PWS as expeditiously as possible,” says Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics.
FPWR will continue to provide the FDA with the patient perspective, emphasizing the tremendous unmet medical need in the PWS population. “Individuals with PWS face enormous challenges, with no currently approved treatments for the most difficult aspects of the disorder. Our community is focused on supporting the advancement of safe and effective treatments for PWS. We appreciate Soleno’s commitment to PWS and look forward to continued conversations with FDA” says Theresa Strong, Director of Research Programs at FPWR.
Read the full press release below.
REDWOOD CITY, Calif., March 08, 2021 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today provided an update following recent interactions with the U.S. Food and Drug Administration (FDA) regarding the development of once-daily DCCR (diazoxide choline) extended release tablets for the treatment of Prader-Willi Syndrome (PWS).
Subsequent to the previously disclosed Type C meeting with the FDA on November 12, 2020 regarding the potential adequacy of data from studies with DCCR to support a New Drug Application (NDA) for the treatment of PWS, Soleno submitted additional analyses to the FDA from the Company’s Phase 3 trial, DESTINY PWS (C601). These data were from study visits that were completed prior to the significant disruptions caused by the COVID-19 pandemic. The data analyses showed statistically significant changes for DCCR compared to placebo in the primary and key secondary endpoints. Following its review of the data submitted by Soleno, the FDA informed the Company on March 5, 2021 that an additional controlled clinical trial will be necessary to support an NDA submission for DCCR in PWS.
“We intend to continue the dialogue with the FDA to ensure that DCCR is approved for individuals with PWS as expeditiously as possible,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics. “Based on the totality of data generated to date, we remain confident in DCCR’s potential to address the unmet need for a safe and effective treatment option for PWS patients. We are currently evaluating the appropriate next steps for our DCCR program.”
About DCCR (Diazoxide Choline) Extended-Release Tablets
DCCR is a novel, proprietary extended-release dosage form containing the crystalline salt of diazoxide and 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 1 clinical studies in healthy volunteers and three completed Phase 2 clinical studies, one of which was in PWS patients. In the PWS Phase 3 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, DCCR extended-release tablets, a once-daily oral tablet for the treatment of Prader-Willi Syndrome (PWS), is currently being evaluated in a Phase 3 clinical development program. For more information, please visit www.soleno.life.