The FDA Has Granted Orphan Status to a Potential Narcolepsy Drug

The United States Food and Drug Administration has awarded Orphan Drug Designation to an investigational drug for narcolepsy, and there are plans to carry out a Phase 2 study to further investigate the drug for this use. For more detailed information about this, you can read the source press release here, at Globe Newswire.

About Narcolepsy

Narcolepsy is a serious neurological condition that affects peoples wake and sleep cycles. People with narcolepsy may find it difficult to stay awake throughout the day, or suddenly fall asleep at inappropriate times. Many people with the condition experience sleep paralysis (being unable to move when waking or falling asleep), disrupted sleep, and cataplexy (temporarily losing muscle control). Narcolepsy can impact many aspects of patients’ daily lives, and advancing treatments is an important area of research.

About the Drug

Axsome Therapeutics is developing an investigational drug as a potential new treatment for patients with narcolepsy. Officially called AXS-12, the drug is a norepinephrine reuptake inhibitor that can be taken orally.

Orphan Drug Status

A spokesperson for Axsome said that they are “very pleased” to have received Orphan Drug status. The designation is designed to encourage the development of new treatments for rare conditions, and gives the developers several benefits, including a seven-year period of marketing exclusivity and tax credits.

Future Research

The company plans to carry out more studies into AXS-12 to further investigate how effective and safe it is as a treatment. A Phase 2 study is planned to begin later this year, with results expected around the first half of 2019.


Anna Hewitt

Anna Hewitt

Anna is from England and recently finished her undergraduate degree. She has an interest in medicine and enjoys writing. In her spare time she likes to cook, hike, and hang out with cats.

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