The landscape of applications for various cannabis compounds is broad and the list of potential cannabis-based therapeutics is lengthy. The extent and the rate at which cannabis therapeutics progress towards approved use depends on a number of factors. These include research and the implementation of well-controlled clinical studies to examine efficacy and toxicity of potential formulations. As well, the development and regulation of standard methods for the production, isolation, and testing of compounds for therapeutic use.
There exists a host of potential therapeutics in various stages of development that suffer from either the lack of sufficient research and clinical data, the absence of distinct production and testing guidelines, or both. The FDA has, however, licensed several cannabinoid-based drugs and have fast tracked others, both natural and synthetic, offering some relief and promise.
Dronabinol (Marinol) is a synthetic delta-9-THC FDA approved drug to treat nausea and vomiting associated with chemotherapy and to stimulate appetite in advanced AIDS patients. A synthetic analog of delta-9-THC, nabilone (Cesamet), is approved for similar indications. The FDA fast tracked drug Sativex (nabiximols) is the world's first plant-derived cannabinoid prescription drug approved for the treatment of spasticity due to multiple sclerosis.
In response to urgent needs, an investigational new drug Epidiolex (concentrated CBD oil) has been developed for use as an anti-seizure medication specifically for Dravet and Lennox-Gastaut syndromes. A main difference in these latter examples is the compounds are natural in origin and not synthetic. If approved, Epidiolex would join Sativex in a promising line of botanical pharmaceuticals to reach approved status. It would be the first plant-based CBD drug as well.
GW Pharma, the company behind Epidiox, has traveled an interesting and at times seemingly impassable road en route to FDA approval. The company went public in early 2013, selling 3.22 M shares at an IPO share price of $90. The company endured rumors of a takeover as stocks soared in response to impending Phase Three clinical trial data in Q3 of 2016. Despite a wealth of private and public investment funds to support development and testing along the way, the timeline for filing the FDA new drug application (NDA) as been pushed back several times, and is currently hanging in suspense at the time of this article.
Despite the uncertainty and angst that has surely clouded the investment arena, the outlook appears promising with four separate Phase 3 trials to support the NDA. It is a matter of time before the drug may be implemented for use with a larger group of patients, many of which are desperate and devoid of alternative therapies. Although the initial indication is for a rare form of epilepsy affecting only 30,000 children and adults in the US, the groundbreaking potential is significant just the same.
Of course there are many moving parts in play that have and can continue to impact progress in a negative way. For instance, a recent delay in the process was reportedly due to the construction and on-boarding of a new extraction facility. Nonetheless, approval would add significant support to the evidence that plant-based cannabis therapeutics are viable as both effective and safe therapeutics. This in turn may have an positive effect on the medical marijuana field as a whole, whose legal status remains complicated to say the least.