Soleno’s Investigational Drug for Rare Genetic Disorder Shows Promise

Soleno Therapeutics has released data from a late-stage study evaluating controlled-release diazoxide choline tablets for patients suffering from Prader-Willi Syndrome (PWS), a rare genetic disorder causing obesity, impaired intellect and shortness of height.

The study in 127 PWS patients four years or older across 29 sites in the U.S. and UK saw a reduction in hyperphagia, an overeating disorder, and a significant reduction in fat mass following 13 weeks of treatment.

PWS patients can be treated with growth promoters, but there are currently no approved therapies to treat the hyperphagia, metabolic function or behavioral aspects of the disorder.

Diazoxide choline has received an Orphan Drug designation for the treatment of PWS in the U.S. and EU, and a Fast-Track designation in the U.S.