On Friday, August 2nd, the PWS Advocacy Coalition submitted a petition to the U.S. Food and Drug Administration (FDA), requesting the filing and priority review of the New Drug Application (NDA) for Soleno Therapeutics’ drug, DCRR. The petition signed by 14,271 members of the PWS community, was sent to FDA’s officials Drs. Tiffany Farchione (Psych Division Director), Dr. Teresa Buracchio (Office of Neuroscience Director), Dr. Peter Stein (Office of New Drugs Drector), and Dr. Patrizia Cavazzoni (CDER Director).
The full petition, along with more than 100 pages of comments, provides a compelling patient voice for the FDA to consider. While we anticipate the FDA will acknowledge the submission of the petition, we do not expect the FDA to comment publicly due to their rules making the review of NDAs “commercial, confidential information.”
Soleno Therapeutics submitted their New Drug Application to the FDA for the approval of DCCR on June 28th, 2024. The FDA has a 60-day filing review period to determine whether the NDA is accepted for review. Soleno has requested a Priority Review of the NDA, which would provide a target review period of six months by the FDA after the NDA has been accepted.
The collection of over 14,000 signatures in support of DCCR’s review and approval is a tremendous accomplishment for the PWS community. We will continue to share updates and details of the FDA’s review of DCCR as they become available.
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. Diazoxide choline has received Orphan Drug Designation for the treatment of PWS in the U.S. and E.U., and Fast Track and Breakthrough Designations in the U.S.
