Can tilorisen (Harcos) cure narcolepsy? Sharing of real patient efficacy data

Pitolisant (brand name Wakix®) is an oral histamine H3 Receptor antagonists/inverse agonists are mainly used for the treatment of adult narcolepsy (narcolepsy) with or without cataplexy (cataplexy). It improves the symptoms of excessive daytime sleepiness and cataplexy by enhancing the release of histamine in the brain and increasing the state of wakefulness. It should be noted that tilorisen can relieve symptoms, but there is currently no cure for narcolepsy, so it cannot be called a "cure."

Clinical data show that patients treated with tilorisen had higher daytime sleepiness scores (Epworth Sleepiness Scale, The average decrease in ESS) was about 6 points, while the placebo group only decreased by about 2 points. About 70% of patients met the efficacy response standard (ESS decreased by ≥3 points). In terms of cataplexy attacks, research shows that the number of weekly attacks can be reduced from an average of about 9 times at baseline to about 2 times. The reduction in the control group was significantly lower than that in the treatment group. This suggests that tilorisen has high clinical effectiveness in controlling somnolence and cataplexy.

Despite its efficacy, tilorisen has limitations. It cannot repair the underlying pathology caused by a lack of orexin neurons in narcolepsy patients and therefore cannot completely cure the disease. Some patients may need to adjust the regimen or discontinue the drug due to insufficient efficacy or intolerance to the drug. Additionally, long-term efficacy data in children and patients with comorbidities remain limited and need to be evaluated with caution.

During use, patients should follow the doctor's instructions and take the medicine regularly every day. Usually, improvement in daytime sleepiness can be observed in the 2–3 weeks, and the number of cataplexy is significantly reduced in the 2–5 weeks. Requires regular monitoringESSscore, frequency of cataplexy, and side effects such as headache, insomnia, anxiety, and nausea. At the same time, good sleep hygiene and life management should be combined to achieve the best therapeutic effect. In summary, tilorisen is an effective drug for relieving symptoms of narcolepsy, but the primary goal should be to improve symptoms rather than cure the disease.

Reference materials:https://www.drugs.com/