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HARMONY BIOSCIENCES ANNOUNCES PLANS TO INITIATE PHASE 3 REGISTRATIONAL STUDY OF PITOLISANT IN PRADER-WILLI SYNDROME FOLLOWING POSITIVE END-OF-PHASE 2 MEETING WITH THE U.S. FOOD AND DRUG ADMINISTRATION
Phase 3 registrational study design to include children, adolescents and adults Study initiation planned for Q4 2023 in pursuit of a potential new indication
About this update from Harmony Biosciences Holdings, Inc.
[{"type":"text","content":"Phase 3 registrational study design to include children, adolescents and adults\n Study initiation planned for Q4 2023 in pursuit of a potential new indication\n\n\nPLYMOUTH MEETING, Pa., July 20, 2023 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. (\"Harmony\") (Nasdaq: HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases, has successfully completed an End-of-Phase 2 meeting with FDA regarding its clinical development plan evaluating pitolisant as a potential treatment for excessive daytime sleepiness (EDS) in patients ages six and older with Prader-Willi syndrome (PWS). Harmony plans to initiate a Phase 3 registrational study in the fourth quarter of 2023. \n\n \n \n \n \n \n \n\n \nBased on a positive signal observed from the Phase 2 proof-of-concept study, Harmony aligned with the FDA on the proposed Phase 3 study design elements to support further investigation of pitolisant for children, adolescents and adults with PWS experiencing EDS. There is currently no FDA-approved treatment for EDS in this patient population.\n\"We are pleased with the outcome of our End-of-Phase 2 meeting with the FDA as we prepare to initiate our Phase 3 registrational study, which aims to further investigate the efficacy and safety of pitolisant as a potential treatment for excessive daytime sleepiness in individuals with Prader-Willi syndrome,\" said Kumar Budur, MD, Chief Medical Officer at Harmony Biosciences. \"Building upon the encouraging data obtained from our Phase 2 signal detection study, we remain committed to advancing our development program for pitolisant in pursuit of a new indication in people with PWS, given the high unmet medical need in this population.\"\nThere are currently 15,000 – 20,000 people in the US living with PWS, a genetic condition often diagnosed in childhood. More than half of patients experience EDS.\nPitolisant is marketed as WAKIX® in the U.S. and is FDA approved to treat EDS or cataplexy in adult patients with narcolepsy. Pitolisant is not approved for use in patients with PWS and is currently being evaluated as an investigational agent in this patient population.\nAbout Prader-Willi SyndromePWS is an orphan/rare, genetic neurological disorder with many of the symptoms resulting from hypothalamic dysfunction. The hypo...