Soleno Therapeutics, Inc. (NASDAQ: SLNO) announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for DCCR (diazoxide choline) extended-release tablets for the treatment of Prader-Willi syndrome (PWS) and granted it Priority Review. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of December 27, 2024.
Regulatory Milestone for DCCR
The FDA's decision to grant Priority Review to DCCR underscores the urgent need for new treatments for PWS, a rare genetic disorder affecting approximately 1 in every 15,000 live births. The FDA review division has indicated that an advisory committee meeting does not appear necessary at this time, though this remains under consideration.
Financial Position and Market Launch Preparations
"In the third quarter of 2024, we achieved a major milestone with FDA acceptance of our NDA seeking approval of DCCR for the treatment of PWS," said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics. "Our dialogue with the FDA has been productive to date, and we look forward to continued collaboration as the review process advances. As we look towards potential approval of DCCR, our commercial team continues to prepare for our planned U.S. market launch. Our strong balance sheet supports successful execution of a launch and the delivery of a transformative therapy to people living with PWS, if approved."
Soleno reported having $284.7 million in cash, cash equivalents, and marketable securities as of September 30, 2024, which the company believes is sufficient to support the potential launch of DCCR.
About Prader-Willi Syndrome
PWS is characterized by hyperphagia, a chronic feeling of insatiable hunger, leading to severe obesity and related complications. Additional characteristics include behavioral problems, cognitive disabilities, low muscle tone, and developmental delays. Currently, there are no approved therapies to treat the hyperphagia or the associated metabolic, cognitive, and behavioral aspects of PWS.
About DCCR
DCCR is a novel, proprietary extended-release formulation of diazoxide choline, designed for once-daily oral administration. Diazoxide has been used for decades in various rare diseases, but DCCR is specifically formulated for PWS. Clinical data from Phase 1, Phase 2, and Phase 3 studies suggest that DCCR can address hyperphagia and other symptoms of PWS, such as aggressive behaviors and metabolic parameters. DCCR has been granted Orphan Drug Designation in the U.S. and E.U., as well as Fast Track and Breakthrough Designations in the U.S.
