New drug pitolisant tested for safety in rare genetic disorder
Disease control
ENROLLING_BY_INVITATION
This phase 3 trial is testing the safety of pitolisant in 150 people with Prader-Willi syndrome who have already taken the drug in a previous study. Participants will receive pitolisant tablets and be monitored for side effects. The goal is to see if pitolisant is safe for long-t…
Phase: PHASE3 • Sponsor: Harmony Biosciences Management, Inc. • Aim: Disease control
Last updated Jun 27, 2026 11:02 UTC