FDA Grants Breakthrough Therapy to Soleno’s Prader-Willi Syndrome Drug

Rare Daily Staff

Rare Daily Staff

The U.S. Food and Drug Administration has granted Breakthrough Therapy designation to Soleno Therapeutics’ diazoxide choline for the treatment of adults and children ages 4 years and older with genetically confirmed Prader-Willi syndrome who have hyperphagia.

The designation reflects the Agency’s determination that, based on an assessment of the preliminary data from the phase 3 clinical development program DCCR extended-release tablets may demonstrate substantial improvement on a clinically significant endpoints over available therapies.

“The granting of Breakthrough Therapy designation, the first for a drug being developed for the treatment of PWS, marks another important milestone for our DCCR clinical development program,” said Anish Bhatnagar, CEO of Soleno Therapeutics. He said the company expects to apply for approval of the drug to the FDA in mid-2024.

Prader-Willi syndrome (PWS) causes chronic and life-threatening feelings of intense, persistent hunger, food pre-occupation, extreme drive to seek food and consume food that severely diminish the quality of life for patients with PWS and their families. Additional characteristics include behavioral problems, cognitive disabilities, low muscle tone, short stature, the accumulation of excess body fat, developmental delays, and incomplete sexual development. Hyperphagia can lead to significant morbidities and mortality. There are currently no approved therapies to treat the hyperphagia/appetite, metabolic, cognitive function, or behavioral aspects of the disorder.

DCCR is a novel, proprietary extended-release dosage form containing diazoxide choline, the crystalline salt of diazoxide, and is administered once-daily. 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 patients 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.

The FDA’s Breakthrough Therapy Designation is intended to expedite the development and review of drugs in the U.S. that are intended to treat a serious condition, when preliminary clinical evidence indicates the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). With Breakthrough Therapy designation, FDA provides intensive guidance and organizational commitment involving senior managers in a proactive, collaborative, cross-disciplinary review, and may also allow for priority review and other actions to expedite review.