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Label indication WAKIX is indicated for the treatment of excessive daytime sleepiness (EDS) or cataplexy in patients 6 years of age and older with narcolepsy. WAKIX is a histamine-3 (H3) receptor antagonist/inverse agonist indicated for the treatment of excessive daytime sleepiness (EDS) or cataplexy patients 6 years of age and older with narcolepsy ( 1 ) FDA label
Also known as Pitolisant Oral Tablet, Pitolisant oral tablets, Open-label Pitolisant, Pitolisant tablet, HBS-101
Pitolisant · 7 trials · 5 indications
A treatment-emergent adverse event (TEAE) is any adverse event reported during treatment with study drug in this study and up to 30 days after final dose of study drug, or any worsening of a pre-existing condition reported during treatment with study drug and up to 30 days after final dose of study drug.
The PROMIS-SRI item bank consists of 13 items with a 5-point rating scale.
Incidence of adverse events (AEs)
Change from Baseline in Epworth Sleepiness Scale (ESS) score The score of the Epworth Sleepiness Scale ranges from 0 to 24. A decrease in score represents an improvement in excessive daytime sleepiness.
The Epworth Sleepiness Scale (ESS) is an 8-item, 4-point rating scale that asks participants to rate the likelihood of falling asleep over the past week. The rating scale ranges from 0 (no chance of dozing) to 3 (high chance of dozing). The total score is calculated by summing the item level ratings. Total scores range from 0 to 24. Higher point totals represent more severe daytime sleepiness.
The score of the DSS ranges from 0 to 15. A decrease in the DSS score represents an improvement in EDS.
| Arm | Type | Description |
|---|---|---|
| Pitolisant | EXPERIMENTAL | All participants receive pitolisant administered orally once daily in the morning upon awakening. |
| Double-Blind Treatment Period Pitolisant | EXPERIMENTAL | Pitolisant tablets administered once daily in the morning upon wakening |
| Double-Blind Treatment Period Placebo | PLACEBO_COMPARATOR | Matching placebo administered tablets once daily in the morning upon wakening |
| Open-Label Extension Period Pitolisant | OTHER | Pitolisant tablets administered once daily in the morning upon wakening |
| Double-blind Pitolisant | ACTIVE_COMPARATOR | Double-blind pitolisant administered once daily in the morning upon wakening for 4 weeks during the Double-Blind Randomized Withdrawal Phase |
| Double-blind Placebo | PLACEBO_COMPARATOR | Matching placebo administered once daily in the morning upon wakening for 4 weeks during the Double-Blind Randomized Withdrawal Phase |
| Open-label Pitolisant | EXPERIMENTAL | Open-label pitolisant administered once daily in the morning upon wakening for 8 weeks during the Open-Label Phase |
| Higher dose pitolisant | ACTIVE_COMPARATOR | Double-Blind Treatment Phase: Week 1: 8.9 mg pitolisant administered once daily in the morning; Week 2: 17.8 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 35.6 mg pitolisant administered once daily in the morning. |
| Lower dose pitolisant | ACTIVE_COMPARATOR | Double-Blind Treatment Phase: Week 1: 4.45 mg pitolisant administered once daily in the morning; Week 2: 8.9 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 17.8 mg pitolisant administered once daily in the morning |
| Placebo | PLACEBO_COMPARATOR | Double-Blind Treatment Phase: Week 1: Matching placebo tablets; Week 2: Matching placebo tablets; Weeks 3 through 11: Matching placebo tablets |
| Double-Blind Treatment Phase Lower Dose Pitolisant | ACTIVE_COMPARATOR | Pediatric patients (6 to less than 12 years of age): Week 1: 4.45 mg pitolisant administered once daily in the morning; Week 2: 8.9 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 8.9 mg pitolisant administered once daily in the morning. Adolescent patients (12 to less than 18 years of age): Week 1: 4.45 mg pitolisant administered once daily in the morning; Week 2: 8.9 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 13.35 mg pitolisant administered once daily in the morning. Adult patients (18 to 65 years of age): Week 1: 4.45 mg pitolisant administered once daily in the morning; Week 2: 8.9 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 17.8 mg pitolisant administered once daily in the morning. |
| Double-Blind Treatment Phase Higher Dose Pitolisant | ACTIVE_COMPARATOR | Pediatric patients (6 to less than 12 years of age): Week 1: 4.45 mg pitolisant administered once daily in the morning; Week 2: 8.9 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 17.8 mg pitolisant administered once daily in the morning. Adolescent patients (12 to less than 18 years of age): Week 1: 8.9 mg pitolisant administered once daily in the morning; Week 2: 17.8 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 26.7 mg pitolisant administered once daily in the morning. Adult patients (18 to 65 years of age): Week 1: 8.9 mg pitolisant administered once daily in the morning; Week 2: 17.8 mg pitolisant administered once daily in the morning; Weeks 3 through 11: 35.6 mg pitolisant administered once daily in the morning. |
| Double-Blind Treatment Phase Placebo | PLACEBO_COMPARATOR | Pediatric patients (6 to less than 12 years of age): Week 1: Matching placebo tablets; Week 2: Matching placebo tablets; Weeks 3 through 11: Matching placebo tablets Adolescent patients (12 to less than 18 years of age): Week 1: Matching placebo tablets; Week 2: Matching placebo tablets; Weeks 3 through 11: Matching placebo tablets Adult patients (18 to 65 years of age): Week 1: Matching placebo tablets; Week 2: Matching placebo tablets; Weeks 3 through 11: Matching placebo tablets |
| Pitolisant Delayed-release: Fasted then Fed | EXPERIMENTAL | Participants will receive 35.6 milligrams (mg) pitolisant delayed-release under fasted conditions. After a washout period of at least 10 days, participants will then receive 35.6 mg pitolisant delayed-release following a low-fat meal. |
| Pitolisant Delayed-release: Fed then Fasted | EXPERIMENTAL | Participants will receive 35.6 mg pitolisant delayed-release following a low-fat meal. After a washout period of at least 10 days participants will then receive 35.6 mg pitolisant delayed-release under fasted conditions. |
| Name | Type | Description |
|---|---|---|
| Pitolisant | DRUG | * Pitolisant 4.45 mg tablets * Pitolisant 17.8 mg tablets |
| Pitolisant tablet | DRUG | Pitolisant tablet |
| Placebo tablet | OTHER | Placebo tablet |
| Open-label Pitolisant | DRUG | Pitolisant 4.45 mg tablets: white, round, plain, biconvex film-coated tablet, 3.7 millimeters (mm) in diameter. Each tablet contains 5 mg of pitolisant hydrochloride equivalent to 4.45 mg of pitolisant. Pitolisant 17.8 mg tablets: white, round, plain, biconvex film-coated tablet, 7.5 mm in diameter. Each tablet contains 20 mg of pitolisant hydrochloride equivalent to 17.8 mg of pitolisant. |
| Double-blind Placebo | DRUG | Matching placebo tablets will be provided for each strength of active pitolisant film-coated tablets. |
| Double-blind Pitolisant | DRUG | Pitolisant 4.45 mg tablets: white, round, plain, biconvex film-coated tablet, 3.7 mm in diameter. Each tablet contains 5 mg of pitolisant hydrochloride equivalent to 4.45 mg of pitolisant. Pitolisant 17.8 mg tablets: white, round, plain, biconvex film-coated tablet, 7.5 mm in diameter. Each tablet contains 20 mg of pitolisant hydrochloride equivalent to 17.8 mg of pitolisant. |
| Pitolisant Oral Tablet | DRUG | Pitolisant 4.45 mg tablets: white, round, plain, biconvex film-coated tablet, 3.7 mm in diameter. Each tablet contains 5 mg of pitolisant hydrochloride equivalent to 4.45 mg of pitolisant. Pitolisant 17.8 mg tablets: white, round, plain, biconvex film-coated tablet, 7.5 mm in diameter. Each tablet contains 20 mg of pitolisant hydrochloride equivalent to 17.8 mg of pitolisant. |
| Placebo oral tablet | DRUG | Matching placebo tablets will be provided for each strength of active pitolisant film-coated tablets. |
| Pitolisant oral tablets | DRUG | Pitolisant 4.45 mg or 17.8 mg tablets |
| Pitolisant delayed-release | DRUG | Delayed-release tablets |
Inclusion Criteria: Each participant must meet the following criteria to be enrolled in this study: 1. Ability to provide voluntary, written informed consent (participant, if applicable, or parent\[s\]/legal guardian\[s\]) and, where applicable, voluntary, written assent (participant, as appropria...
Pitolisant is a small molecule being developed by Harmony Biosciences Holdings, Inc. (ticker HRMY) for neurological sleep and neurological disorders. It is in Phase 3 clinical development for idiopathic hypersomnia, myotonic dystrophy 1, and Prader-Willi Syndrome. Pitolisant is also known as Pitolisant oral tablets or Pitolisant tablet.
Pitolisant is being studied for idiopathic hypersomnia, myotonic dystrophy 1, and Prader-Willi Syndrome. Phase 3 trials have enrolled adults with idiopathic hypersomnia and excessive daytime sleepiness, and pediatric and adult participants with Prader-Willi Syndrome. The drug is investigational and is not approved for these indications.
Pitolisant targets HRH3, the histamine H3 receptor, and acts as an inverse agonist. By targeting HRH3, the drug is designed to modulate histamine signaling in the central nervous system. This mechanism is being evaluated in Phase 3 studies for idiopathic hypersomnia, myotonic dystrophy 1, and Prader-Willi Syndrome.
Pitolisant is developed by Harmony Biosciences Holdings, Inc., which trades under the ticker HRMY. The company is running Phase 3 clinical programs for the drug in idiopathic hypersomnia, myotonic dystrophy 1, and Prader-Willi Syndrome.
Pitolisant is in Phase 3 clinical development. The program includes completed Phase 3 trials in adult idiopathic hypersomnia and ongoing Phase 3 trials in Prader-Willi Syndrome. Pitolisant is investigational for the indications under study and has not been approved for them.
Pitolisant has been studied in Phase 3 trials including NCT05156047 and NCT05458128 in adult idiopathic hypersomnia, both completed. Ongoing Phase 3 studies include NCT06366464, which is recruiting participants with Prader-Willi Syndrome across multiple countries, and NCT07219485, which is enrolling by invitation in the United States.
Yes, Pitolisant oral tablets, Pitolisant tablet, and Open-label Pitolisant all refer to the same drug, Pitolisant. These names describe the oral tablet formulation and open-label study use of the same small molecule developed by Harmony Biosciences Holdings, Inc. (HRMY) for idiopathic hypersomnia, myotonic dystrophy 1, and Prader-Willi Syndrome.