- GW Pharmaceuticals and its U.S. subsidiary Greenwich Biosciences on Monday won approval from the Food and Drug Administration for its cannabis-based drug Epidiolex.
- The drug was OK'd for seizures related to Dravet syndrome and Lennox-Gastaut syndrome, two rare forms of epilepsy, in patients older than two. There are about 36,000 patients total for the two syndromes in the U.S.
- Epidiolex is the first drug derived from the marijuana plant to be approved by the FDA. The agency was quick to note in its statement that "[cannabidiol] is a chemical component of the Cannabis sativa plant ... However, CBD does not cause intoxication or euphoria (the "high") that comes from tetrahydrocannabinol (THC). It is THC (and not CBD) that is the primary psychoactive component of marijuana."
GW Pharma CEO Justin Gover told BioPharma Dive in an interview that the approval was the culmination of nearly 20 years of work and that it would be a "new era" for the U.K.-based biotech, which will be transitioning to a commercial-stage company for the first time. GW has been prepping for this moment for several years, with Gover himself moving to the U.S. three years ago to oversee the commercial build-out.
While the approval is a landmark, it was not entirely unexpected. In April, an FDA Advisory Committee voted unanimously in favor of approving Epidiolex (cannabidiol), calling its abuse-potential "low."
Currently, Epidiolex is considered a Schedule I drug by the Drug Enforcement Administration. Now that Epidiolex has been approved, GW needs to wait for rescheduling of the compound for them to bring it to market — the DEA has 90 days to issue its decision. While the FDA has no say in the process, it does make a recommendation to its enforcement counterpart.
Gover said in an interview that the company wants to make no presumptions about the scheduling, but expects — based on the data — that Epidiolex will be either Schedule IV or Schedule V. Gover explained that a Schedule V designation would largely be "around auditing and accounting" and "would have very little visibility to the physician or the patient." Most epileptic medicines are Schedule V. Other examples of drugs in this class include cough medicine with minimal amounts of codeine.
GW hopes to launch the drug in the fall of 2018 with 70 sales reps that will target 4,500 to 5,000 neurologists and epileptologists.
The company currently manufactures the product in the U.K., where it is based. It grows the specific strain of the marijuana plant at the U.K.'s largest "glasshouse" in a process that lowers the likelihood of a high and increases the amount of CBD. The product is then purified into a crystallized form of CBD that is highly regulated and quality-controlled.
"[I]t's also important to note that this is not an approval of marijuana or all of its components. This is the approval of one specific CBD medication for a specific use. And it was based on well-controlled clinical trials evaluating the use of this compound in the treatment of a specific condition. Moreover, this is a purified form of CBD. It's being delivered to patients in a reliable dosage form and through a reproducible route of delivery to ensure that patients derive the anticipated benefits," wrote FDA Commissioner Scott Gottlieb in a statement.
Epidiolex will be dispensed with a medication guide that spells out certain risks, including thoughts about suicide, attempts to commit suicide, feelings of agitation, new or worsening depression, aggression and panic attacks, as well as the risk of liver injury.
GW received a rare pediatric disease priority review voucher. PRVs are given out as an incentive to support the development of drugs for rare disease in children. GW can use the voucher to expedite the approval of its next compound or can sell the PRV.