Pitolisant hydrochloride is under clinical development by Harmony Biosciences and currently in Phase III for Hypersomnia. According to GlobalData, Phase III drugs for Hypersomnia have a 75% phase transition success rate (PTSR) indication benchmark for progressing into Pre-Registration. GlobalData’s report assesses how Pitolisant hydrochloride’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Pitolisant hydrochloride overview

Pitolisant hydrochloride (Wakix, Ozawade) is a central nervous system drug. It is formulated as film-coated tablets for oral route of administration. Wakix is indicated in adults for the treatment of narcolepsy with or without cataplexy. Ozawade is indicated to improve wakefulness and reduce excessive daytime sleepiness (EDS) in adult patients with obstructive sleep apnoea (OSA) whose EDS has not been satisfactorily treated by, or who have not tolerated, OSA primary therapy, such as continuous positive airway pressure (CPAP).

It is under development for the treatment of obstructive sleep apnea, excessive diurnal sleepiness in patients with moderate to severe obstructive sleep apnoea (OSA) syndrome, obstructive sleep apnea and narcolepsy with or without cataplexy in adults and pediatrics and idiopathic hypersomnia. It is also under development for prader-willi syndrome (PWS), fatigue, cognitive impairment and myotonic dystrophy. It was also under development for the treatment of bipolar depression and cognitive-enhancing effects in schizophrenia or schizoaffective disorder and alcohol use disorder and Parkinson's disease.

Harmony Biosciences overview

Harmony Biosciences is a biopharmaceutical company that discovers, develops and commercializes therapies for neurological indications. The company offers WAKIX medication for the treatment of narcolepsy, a neurologic disorder of sleep-wake state instability in adults. It is investigating Pitolisant, a histamine-3 receptor (H3R) antagonist-based program to treat idiopathic hypersomnia, Prader-Willi syndrome and myotonic dystrophy; and HBS-102, a melanin-concentrating hormone receptor 1 (MCHR1) antagonist for Prader-Willi syndrome. Harmony Biosciences is headquartered in Plymouth Meeting, Pennsylvania, the US.

For a complete picture of Pitolisant hydrochloride’s drug-specific PTSR and LoA scores, buy the report here.


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.