Treatment for Cataplexy and Narcolepsy Gets Designations From the FDA

According to a story from, the company Harmony Biosciences, Inc., has recently announced that the U.S. FDA has given Fast Track and Breakthrough Therapy designations for its experimental therapy pitolisant. This product is in development for the treatment of narcolepsy and cataplexy. These designations are indications of the FDA’s increased interest in the development of pitolisant.
Narcolepsy is a long term disorder in which the ability of a patient to regulate their sleep-wake cycle is significantly affected. The primary symptom of narcolepsy are episodes of extreme daytime sleepiness that can vary in length from just a few seconds to several minutes. Another symptom that often occurs alongside narcolepsy is cataplexy, which are episodes of sudden loss of muscle strength. These occur in about 70 percent of narcolepsy patients. Episodes of muscle weakness can be triggered by strong emotional reactions. Most people with narcolepsy get almost normal amounts of sleep, but it is of low quality and they are in a constant state of deprivation. They may also experience hallucinations and sleep paralysis. The cause of narcolepsy is unknown, though there is family history in about ten percent of cases. Treatments include stimulants and antidepressants, though it can take months before symptoms stabilize. To learn more about narcolepsy and cataplexy, click here.

Breakthrough Therapy designation was granted to pitolisant for the treatment of cataplexy in narcolepsy patients and Fast Track designation was granted for the same indication as well as the treatment of excessive daytime sleepiness in patients with narcolepsy. Breakthrough Therapy designation is reserved for treatments that display the potential to significantly improve outcomes over currently available therapies for a serious condition or disease. Fast Track designation is reserved for treatments that aim to fulfill a serious unmet medical need or that demonstrate a significant advantage over currently available treatments. Recipients of these designations are eligible for close collaboration with the FDA, accelerated approval and priority review, and assistance from the agency in drug development.

The drug previous received orphan drug designation from the FDA as well. If approved, pitolisant would be the first new treatment approved for excessive daytime sleepiness and cataplexy in narcolepsy patients in over ten years.

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