INFORMATION INTENDED FOR GLOBAL HEALTHCARE PROFESSIONALS

Epidiolex®

GW’s Epilepsy Clinical Program

GW is committed to developing new medicines to treat rare, treatment-resistant epilepsy conditions where there are limited or in some cases, no approved treatment options.

Epidiolex is GW’s lead cannabinoid product candidate and is a proprietary oral solution of pure plant-derived cannabidiol, or CBD. GW’s Epidiolex development is initially concentrating on severe, orphan, early-onset, treatment-resistant epilepsy syndromes including Dravet syndrome, Lennox-Gastaut syndrome (LGS) and Tuberous Sclerosis Complex (TSC).

GW’s Epidiolex development includes two distinct programs:

Pivotal-clinical trial program

  • We have conducted a series of clinical trials designed to obtain safety and efficacy data on our cannabidiol oral solution to provide to the FDA and other regulatory authorities around the world, designed to support approval as a prescription medicine. Initial target indications include Dravet syndrome, Lennox-Gastaut syndrome and Tuberous Sclerosis Complex. In these trials, eligible patients are randomly assigned to receive CBD or placebo added to their current treatment and evaluated over a specific period of time. These trials are “blinded” meaning that patients, families, and physicians do not know which treatment arm they have been assigned.
  • GW’s Phase 3 pivotal trial program for Epidiolex includes two Phase 3 trials in Dravet syndrome, two in LGS, and one in TSC. Three of these trials, one in Dravet syndrome and two in LGS, have concluded. (see GW press releases: 14 March 2016 & 27 June 201626 September 2016).
  • To learn more about GW’s Epidiolex clinical trials please see the ClinicalTrials.gov website here.

FDA-authorized, independent Physician-led program or Expanded Access (which are at times called Compassionate Use programs in some countries) and for which GW supplies CBD* (click here for information on the U.S. Expanded Access Program)

  • The FDA may authorize expanded access programs to facilitate access to investigational drugs for treatment use for patients with a serious or immediately life-threatening disease or condition who lack therapeutic alternatives. This is done through FDA granting Investigational New Drug (IND) applications.
  • The FDA has granted individual patient emergency INDs to physicians as well as INDs to physicians and state programs to treat groups of patients suffering from intractable epilepsy with Epidiolex.
  • The most recent physician-reported data from this Expanded Access Program was presented in December 2015 at the American Epilepsy Society’s annual meeting. Results from 261 patients receiving Epidiolex under these INDs showed promising signals of clinical effect in reducing seizures (link to press release and poster).

Support and Advocacy Organizations

There are a number of organizations which provide invaluable help, information, and support to people living with epilepsy. They are also a useful resource for caregivers, friends, and relatives. The following list includes links to some websites of patient organizations that may be useful.

Physicians, if you are interested in participating in a GW-sponsored clinical trial, please contact GW at: medicalinformation@gwpharm.com