This article is part of an ongoing blog series profiling the 12 new, game-changing drugs predicted to achieve blockbuster status by 2022 in the 2018 edition of Drugs to Watch, the annual industry forecast and analysis from Clarivate Analytics. Read the full Drugs to Watch report here or follow the series for the latest updates.
The 2018 edition of the annual industry forecast and analysis review of Clarivate Analytics, the Drugs to Watch, predicted the market entry of GW Pharmaceuticals’ Epidiolex – an agent that had the potential not just to be a blockbuster, but also the first approved cannabinoid-based anti-epileptic medication. In June 2018, Epidiolex achieved this, with approval in the U.S. for the treatment of seizures associated with two orphan indications, Lennox-Gastaut syndrome (LGS) and Dravet syndrome.1 Furthermore, Epidiolex is the first FDA-approved therapy for Dravet syndrome.
LGS is a form of severe epilepsy and is characterized by multiple types of seizures and intellectual disability. People with LGS begin having frequent seizures in early childhood, usually between the ages of 3 and 5 years. More than three-quarters of affected individuals have tonic seizures, which cause the muscles to stiffen (contract) uncontrollably.2 Dravet syndrome is another severe form of epilepsy. It appears during the first year of life with frequent fever-related seizures.3
Epidiolex’s efficacy data have been compelling in both disease settings. In patients with Dravet syndrome, Epidiolex treatment led to a 39% decrease in the frequency of seizures at 14 weeks, versus 13% for placebo.4 In patients with LGS, a disease highly resistant to treatment, Epidiolex treatment led to a 44% reduction in seizures frequency, versus 22% for placebo.5
While a cannabis-based medicine has previously been approved in Europe, U.S. approval of Epidiolex will pave the way for approval of medical cannabinoids for other indications in the U.S., opening up a large potential market.”
Launch of Epidiolex is expected later this year, once the drug has been rescheduled from its current listing as a Schedule 1 controlled drug.1,6 While a cannabis-based medicine has previously been approved in Europe (GW Pharmaceuticals’ Sativex for multiple sclerosis-related spasticity), U.S. approval of Epidiolex will pave the way for approval of medical cannabinoids for other indications in the U.S., opening up a large potential market. In addition, availability of this cannabinoid-based medication will provide patients, who may previously have used cannabidiol from marijuana dispensaries due to the lack of treatment options, with access to a pure and regulated product that lacks the “high” associated with marijuana.7,8
An MAA for approval of the drug in Europe has been filed, with a decision expected in 1Q19,9 although development is not being pursued in Japan, where the law prevents regulators approving cannabis-based medicines.10 As predicted in March 2018 upon release of the Drugs to Watch, Epidiolex is still expected to become a blockbuster, with Cortellis Consensus Forecast data (source Thomson Reuters I/B/E/S) of $1.083 billion in 2022, rising to $1.273 billion in 2023.