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Also known as Pitolisant tablet
Pitolisant · 6 trials · 4 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 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 |
| 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 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 |
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 an investigational small molecule being developed for myotonic dystrophy type 1, idiopathic hypersomnia, and Prader-Willi syndrome. It is in Phase 3 clinical development for these neurologic conditions. The drug is being studied to address excessive daytime sleepiness and other symptoms associated with these disorders.
Pitolisant is being developed by Harmony Biosciences Holdings, Inc., a biopharmaceutical company traded on NASDAQ under the ticker HRMY. The company is conducting clinical trials to evaluate the drug's safety and efficacy across multiple neurologic indications.
Pitolisant is in Phase 3 clinical development. It has completed Phase 2 trials in Prader-Willi syndrome and myotonic dystrophy type 1, and is currently being evaluated in Phase 3 studies for Prader-Willi syndrome. The drug remains investigational and has not been approved by regulatory authorities.
Pitolisant is being studied in several clinical trials. NCT06366464 is a Phase 3 recruiting study in Prader-Willi syndrome with 134 participants. NCT07219485 is a Phase 3 study enrolling by invitation in the same condition. Completed trials include NCT04257929 in Prader-Willi syndrome and NCT04886518 in myotonic dystrophy type 1.
Yes, Pitolisant tablet is the same drug as Pitolisant. The tablet formulation is the oral dosage form being evaluated in clinical trials. Both names refer to the same investigational small molecule developed by Harmony Biosciences for neurologic conditions.
Pitolisant is a small molecule that targets the histamine H3 receptor, acting as an inverse agonist. By modulating this receptor, it increases histamine release in the brain, which promotes wakefulness and may address excessive daytime sleepiness associated with conditions like Prader-Willi syndrome and myotonic dystrophy type 1.