The American Academy of Neurology has reported promising results in early trials of the drug, Epidiolex, which is 98 percent pure cannabidiol, in treating severe forms of pediatric epilepsy, including Dravet syndrome and Lennox-Gastaut syndrome. In an open-label clinical trial, more than 50 percent of the 137 children and young adults who took the drug saw a decrease in their seizures over a three-month period.
Well-documented anecdotal evidence about the efficacy of cannabis in treating seizures has been for available since at least 1843. What should be particularly encouraging to investors, however, is the more recent involvement of the FDA and the scientific community. For the politically-minded, FDA approval can provide a powerful argument for the rescheduling of marijuana.
Epidiolex Likely to be Available in 2015
The good news for parents and patients is that the drug is being studied under the FDA’s expanded access investigational new drug process. When the results of the open-label trial are approved by the FDA and the Drug Enforcement Administration, patients with serious or immediately life-threatening conditions who have no alternative treatment options may be treated with Epidiolex outside of clinical trials. Epidolex is expected to become available to qualifying individuals later this year.
In the meantime, however, the drug’s maker, GW Pharmaceuticals (NASDAQ: GWPH) is proceeding with the next phase of clinical trials involving a larger sample size and double-blind testing. Double-blind testing will involve the use of a placebo in instances unknown to both patients and doctors and is the FDA’s gold standard for full approval.
Epidiolex is not the only cannabis-based medication to seek FDA approval. Sativex, another GW Pharmaceuticals product, which contains both CBD and THC, has been approved for treatment of muscle spasms associated with multiple sclerosis in 27 countries and is currently waiting for U.S. approval.
The company is also evaluating the potential for cannabinoids in the treatment of Type 2 diabetes, ulcerative colitis, central nervous system disorders, including epilepsy and schizophrenia, cancer and neurodegenerative disease. Marinol, a synthetic THC-based drug, was approved for treatment of nausea and vomiting associated with AIDS and cancer chemotherapy by the FDA in 1985.
A Back Door to Rescheduling
It is the bane of the legal industry’s existence that marijuana is classified as a Schedule I drug, meaning that it is not considered legitimate for medical use. The synthetic THC in Marinol is, however, classified as a Schedule III drug, much like codeine. The logic of the different classification for the naturally-occurring and synthesized chemical has escaped observers for some time.
FDA approval of Epidiolex and Sativex could lend considerable weight to the argument that cannabis-based drugs, whether comprised of THC, CBD or other cannabinoids, do in fact, have a recognized medical use and should thus be reclassified or declassified. It is worth hoping, in any event, that law may come to be informed by science in the future. In the meantime, however, patients with devastating diseases and their parents may have another treatment alternative available in the near future. Investors can watch for further developments on the FDA approval process.