PLYMOUTH MEETING, PA — Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) announced that the U.S. Food and Drug Administration (FDA) recently granted Orphan Drug designation to pitolisant for the treatment of idiopathic hypersomnia (IH).
The FDA’s Orphan Drug designation incentivizes the advancement of promising therapies for rare diseases. Approximately 80,000 people in the U.S. are believed to be affected by IH, with 40,000 currently having been diagnosed. IH is a condition with high unmet medical need.
Harmony is currently evaluating the efficacy and safety of pitolisant in adult patients with IH in the Phase 3 registrational INTUNE study, a double-blind, placebo-controlled, randomized withdrawal study. Topline study results are anticipated in the fourth quarter of 2023 following enrollment completion nine months ahead of plan.
“The FDA’s decision to grant Orphan Drug designation reinforces our belief in pitolisant as a promising therapy for adult patients with IH, with the unique added benefit of it being a non-scheduled, once-daily treatment option working through histamine to improve wakefulness,” said Kumar Budur, M.D., Chief Medical Officer at Harmony Biosciences. “With the completion of enrollment in our INTUNE study nine months ahead of schedule and topline results expected in Q4, this designation is a significant advance in our clinical and commercial development initiatives. We look forward to working closely with the FDA and the broader IH community to address the unmet medical needs of patients living with this condition.”