Harmony Biosciences Receives FDA's Orphan Drug Designation For Pitolisant In Prader-Willi Syndrome

(RTTNews) - Harmony Biosciences Holdings, Inc. (HRMY), Tuesday announced that the U.S. Food and Drug Administration or FDA granted Orphan Drug designation to Pitolisant for the treatment of Prader-Willi syndrome or PWS.

PWS is a rare, genetic neurological disorder, that causes obesity, intellectual disability, low muscle tone, and short stature.

The company stated that the upcoming Phase 3 TEMPO study will assess the safety and efficacy of Pitolisant in treating EDS and behavioral disturbances in PWS. It will be a randomized, double-blind, placebo-controlled trial in patients six years and older with PWS.

The biotechnology company's TEMPO study is expected to start in the first quarter of 2024.

The Orphan Drug designation would provide the company with tax credits for clinical development, a waiver for user fees, and seven years of market exclusivity after drug approval.

In the pre-market activity, Harmony's stock is moving up 0.59 percent, to $34.01 on the Nasdaq.