The U.S. FDA has extended the review period for Soleno Therapeutics' new drug application for DCCR (diazoxide choline) extended-release tablets for the treatment of Prader-Willi syndrome, pushing the PDUFA date to March 27, 2025.
The extension was based on the agency's determination that responses to recent information requests constituted a major amendment to the NDA. The FDA did not cite any safety, efficacy or manufacturing concerns in its correspondence.
Prader-Willi Syndrome is a rare genetic condition usually caused by deletion of a part of chromosome 15 passed down by the father. The hallmark symptom of the 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.
Soleno is pursuing approval of DCCR in individuals four years and older who have hyperphagia. The drug, which has been granted Breakthrough and Fast Track designations in the U.S., as well as Orphan Drug Designation in the U.S. and EU for the treatment of PWS, originally had a PDUFA target action date of December 27, 2024. In October, the FDA Review Division determined that there did not appear to be a need for an advisory committee meeting.
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 individuals in a few rare diseases in infants, children and adults, but is not yet approved for use in PWS.