16. Epidiolex

GW Pharma growing area for CBD Epidiolex
(GW Pharma)

Active ingredient: cannabidiol
Disease: Lennox-Gastaut syndrome and Dravet syndrome
Peak sales estimate: $1 billion
Approved: June 25
Company: GW Pharma

The scoop: With a nod for two rare forms of epilepsy, GW Pharma’s Epidiolex became the first cannabis-based prescription medicine in the U.S. The drug is a purified form of cannabidiol derived from plants that treats seizures associated with Lennox-Gastaut and Dravet syndromes, which tend to appear in childhood, have a higher mortality rate than other types of epilepsy and do not respond to many of the drugs typically used to treat epilepsy. While it is unclear how, exactly, Epidiolex wards off seizures, it doesn’t appear to interact with cannabinoid receptors and does not look to have abuse potential. Despite nabbing the FDA’s OK in June, GW Pharma couldn’t launch the drug until the Drug Enforcement Administration rescheduled Epidiolex, which it did in September. However, the rescheduling of cannabidiol—Epidiolex's active ingredient—is not a blanket decision. Cannabis and other forms of cannabidiol remain classified as Schedule I substances, a category that covers drugs with no therapeutic value and high potential for abuse. — Amirah Al Idrus

16. Epidiolex

Suggested Articles

Bayer CEO Werner Baumann stressed “less and less trust in society" for technology advances as a hurdle for pharma companies working on gene editing.

Kintai Therapeutics' anti-obesity candidate KTX-0200 showed sustained weight loss and improved glucose control and liver health in rodents.

Akcea's triglyceride-fighter beat placebo at lowering blood triglyceride levels in patients who have, or are at risk of, heart disease.