On Monday, June 25, 2018, the United States Food and Drug Administration announced the approval of the first marijuana-based prescription medication in the US. The drug, called Epidiolex, is a plant-derived oral solution of cannabidiol (CBD). CBD, a chemical derived from cannabis, is a cannabinoid that does not produce psychoactive effects when consumed.
Produced by UK-based firm GW Pharmaceuticals PLC,Epidiolex was approved to treat patients aged two and older who suffer from Lennox-Gastaut syndrome and Dravet syndrome, both rare and severe forms of epilepsy that can develop in early childhood. This move also marks the FDA’s first approval of a drug for the treatment of patients with Dravet syndrome.
According to a press release from the FDA, Epidiolex’s effectiveness was studied in three randomized, double-blind, placebo-controlled clinical trials involving 516 patients with either Lennox-Gastaut syndrome or Dravet syndrome. Epidiolex, taken along with other medications, was shown to be effective in reducing the frequency of seizures when compared with placebo.
Because it is a chemical component of the cannabis plant, CBD is currently a Schedule I substance under the Controlled Substances Act. With this approval, there is more news expected to come soon: a reclassification of CBD by the US Drug Enforcement Administration. Once this happens, we will likely see more marijuana-derived medications in the near future.
We did a quick Q+A with our CEO, Rob Davidson, and COO, Jessica Rousset to get their thoughts on this historic move by the FDA and what it means for the industry at large.
- How significant is the Epidiolex approval?
Jessica: The Epidiolex approval is game-changing as it validates, for the first time, the clinical efficacy of a drug derived from the cannabis plant rather than chemically synthesized.
Rob: This approval may accelerate the approval of whole plant extracts containing other types of cannabinoids. There are over 140 cannabinoid molecules in the cannabis plant with potential therapeutic effects, as well as terpenes and flavonoids that have biological activity and could be therapeutically active.
- How does the FDA’s first approval of a CBD product impact the pharmaceutical industry as a whole?
Jessica: This approval highlights the incongruity among federal agencies, where the FDA has now ruled that a cannabis extract is an effective treatment for reducing seizures but the DEA does not recognize any medical benefit for the same product. A meaningful outcome from this approval would be the re-scheduling or de-scheduling of cannabis products by the DEA, consistent with the FDA’s conclusions, and specifically CBD extracts.
- To the best of your knowledge, when do you expect other CBD drugs to achieve FDA approval?
Jessica: The CBD-focused drug pipeline, bio-synthesized, chemically synthesized or whole plant derived is robust with over 90 candidates in development.
Rob: Approval of any active ingredient for a specific indication is predicated on the clinical efficacy and outcome of well controlled studies. Although CBD shows promise in many indications, many have yet to complete phase 3 studies – the ultimate hurdle to approval.
- How many cannabinoid drugs with official FDA approval do you expect to see in the near future?
Rob: While there are many CBD-based drug candidates in the pipeline, we know that only a subset will meet their endpoints and be approved. I would say that we are on our way to at least 6-12 cannabinoid drugs being approved in the next ten years. What I’m most excited about are those other less known cannabinoid molecules that are showing great promise in several indications, including cancer.
- How far along is CURE with its cannabinoid program?
Jessica: Our focus has been on THC and other cannabinoid molecules such as palmitoylethanolamine (PEA). We are developing oral film formulations of these compounds and others to increase their absorption and improve the patient experience. With our partners, we are targeting indications such as sleep apnea and cognitive impairment.