Harmony Biosciences Gets FDA Orphan Designation for Pitolisant in Prader-Willi Syndrome

By Colin Kellaher

Harmony Biosciences Holdings has won orphan-drug designation from the U.S. Food and Drug Administration for its pitolisant drug candidate for the treatment of the genetic disorder Prader-Willi syndrome.

The Plymouth Meeting, Pa., pharmaceutical company said it plans to assess the safety and efficacy of pitolisant in treating excessive daytime sleepiness and behavioral disturbances in patients with Prader-Willi in a Phase 3 registrational study slated to launch during the first quarter.

The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S. and provides for an extended marketing exclusivity period against competition.

Harmony said 15,000 to 20,000 people in the U.S. are living with Prader-Willi, a rare neurological disorder with symptoms that result from dysfunction of the part of the brain that controls sleep-wake state stability and signals that mediate the balance between hunger and satiety.

Write to Colin Kellaher at [e-mail]

(END) Dow Jones Newswires

02-20-24 0834ET