Harmony Biosciences Presents New Analysis Of Clinical Impact For WAKIX® (pitolisant) At The SLEEP 2021 Annual Meeting
Data highlight the clinical effectiveness of WAKIX for excessive daytime sleepiness and cataplexy in adults with narcolepsy
Assessment of the Clinical Impact of Pitolisant on Excessive Daytime Sleepiness and Cataplexy in Adults With Narcolepsy
This poster presents results from a new analysis that evaluated the clinical impact of pitolisant in adults with narcolepsy using NNT and effect size as measures of treatment response based on data from the HARMONY 1 and HARMONY CTP randomized, controlled clinical trials. NNT and effect size are important metrics as they measure the number of patients that need to be treated to achieve a specific outcome for one person (NNT) and the magnitude of drug–placebo difference on outcome measures (effect size). NNT values less than 10 are considered to represent a clinically meaningful difference (with lower values equating to greater efficacy) and thresholds for effect size are 0.2 for a small effect, 0.5 for a medium effect, and 0.8 representing a large effect size, with larger effect sizes signifying greater efficacy.
For improvement in EDS, NNT was based on response defined as an Epworth Sleepiness Scale (ESS) score reduction of ≥3 or final ESS score ≤10 and effect size was based on an analysis of
The results for improvement in EDS were the following:
- NNT was 5 in HARMONY 1 and 3 in HARMONY CTP
- Effect size was 0.61 in HARMONY 1 and 0.86 in HARMONY CTP
The results for reduction in the WRC from HARMONY CTP were the following:
- NNT was 3 for a 50% reduction in the WRC and 4 for a 75% reduction in the WRC
- Effect size was 0.86
"This analysis utilizing NNT and effect size offers a different perspective on the efficacy profile of WAKIX compared to traditional clinical trial endpoints, and is one that is clinically relevant" said Harmony's Chief Medical Officer,
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 neurological 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
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
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