Harmony Biosciences Highlights Publication of WAKIX® (pitolisant) Efficacy Data in Sleep Medicine
The publication, Efficacy of Pitolisant in Patients with High Burden of Narcolepsy Symptoms: Pooled Analysis of Short-Term, Placebo-Controlled Studies reports data pooled from two randomized, placebo-controlled, 7-or 8-week studies in adult patients with narcolepsy, in which pitolisant could be titrated to a maximum dose of 35.6 mg/day. Three independent patient subgroups were analyzed based on the following criteria for high burden of narcolepsy symptoms:
- High burden of EDS as defined by an Epworth Sleepiness Scale (ESS) baseline score ≥16 (n = 118);
- High burden of EDS as defined by a sleep latency ≤8 minutes on a Maintenance of Wakefulness Test (MWT) (n = 105); and,
- High burden of cataplexy as defined by ≥15 cataplexy attacks per week (n = 31).
Change from baseline to the end of treatment was evaluated for pitolisant compared with placebo in each patient subgroup.
The publication reports the following results:
- In the patients with a high burden of EDS as defined by ESS (pitolisant, n=60; placebo, n=58), least-squares mean change from baseline on the ESS was significantly greater for pitolisant (–6.1) compared with placebo (–2.3; p<0.001). In addition, significantly more patients were classified as treatment responders (ESS score reduction ≥3) in the pitolisant group (69 percent) compared with the placebo group (35.1 percent; p=0.001).
- In the patients with a high burden of EDS as defined by the MWT (pitolisant, n=59; placebo, n=46), increase in mean sleep latency on the MWT was significantly greater for pitolisant (6.9 minutes) compared with placebo (3.4 minutes; p=0.017).
- In the patients with a high burden of cataplexy (pitolisant, n=20; placebo, n=11), least-squares mean change in the weekly rate of cataplexy was significantly greater for pitolisant (–14.5; baseline, 23.9; final, 9.4) compared with placebo (–0.1; baseline, 23.1; final, 23.0; p=0.004).
- The adverse event profile from this analysis was consistent with the known safety profile for pitolisant; headache, nausea, and anxiety were the most common adverse events in pitolisant-treated patients.
"Narcolepsy is a chronic, debilitating neurological disorder characterized by sleep-wake state instability that can disrupt daily functioning, especially in patients who experience a high symptom burden," said Harmony's Chief Medical Officer,
Pitolisant is marketed as WAKIX in the
About WAKIX® (pitolisant) Tablets
WAKIX, a first-in-class medication, is approved by the
Important Safety Information
WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation and in patients with severe hepatic impairment. WAKIX is extensively metabolized by the liver and there is a significant increase in WAKIX exposure in patients with moderate impairment.
WAKIX prolongs the QT interval. Avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use of WAKIX in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval.
The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment. WAKIX is not recommended in patients with end-stage renal disease (ESRD).
In the placebo-controlled clinical trials conducted in patients with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and twice placebo) for WAKIX were insomnia (6%), nausea (6%), and anxiety (5%). Other adverse reactions that occurred at ≥2% and more frequently than in patients treated with placebo included headache, upper respiratory infection, musculoskeletal pain, heart rate increased, hallucinations, irritability, abdominal pain, sleep disturbance, decreased appetite, cataplexy, dry mouth, and rash.
Please see the Full Prescribing Information for WAKIX for more information.
Narcolepsy is a rare, chronic, debilitating neurologic disorder of sleep-wake state instability that impacts approximately 165,000 Americans and is primarily characterized by excessive daytime sleepiness (EDS) and cataplexy – its two cardinal symptoms – along with other manifestations of REM sleep dysregulation, which intrude into wakefulness. EDS is the inability to stay awake and alert during the day and is the symptom that is present in all people living with narcolepsy. In most patients, narcolepsy is caused by the loss of hypocretin, a neuropeptide in the brain that supports sleep-wake state stability. This disorder affects men and women equally, with typical symptom onset in adolescence or young adulthood; however, it can take up to a decade to be properly diagnosed.
About Harmony Biosciences
Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare, neurological disorders who have unmet medical needs. For more information on Harmony Biosciences, please visit the company's website: www.harmonybiosciences.com.
Forward Looking Statement
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our product WAKIX®. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: our commercialization efforts and strategy for WAKIX®; the rate and degree of market acceptance and clinical utility of WAKIX®, pitolisant in additional indications, if approved, and any other product candidates we may develop or acquire, if approved; our research and development plans, including our plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreement with
Harmony Biosciences Media Contact:
Harmony Biosciences Investor Contact:
View original content to download multimedia:http://www.prnewswire.com/news-releases/harmony-biosciences-highlights-publication-of-wakix-pitolisant-efficacy-data-in-sleep-medicine-301248750.html