Last week, an FDA Advisory committee unanimously recommended the approval of Epidiolex for the treatment of two rare forms of pediatric epilepsy (Lennox-Gastaut syndrome and Dravet syndrome).
The full report is available from the FDA, while the sponsor’s press release is available here.
The recommendation is based on three Phase III studies, in which Epidiolex reduced seizure frequency when added to current therapy. In fact, the reduction in seizure frequency is quite dramatic.
Eyeballing the tables in the FDA report, it looks like seizure frequency is reduced by ~40%; a significant reduction when one considers these patients experience multiple seizures per day.
Adverse events were mild, and limited to upticks in liver function, and infection (especially pneumonia).
The FDA is not required to follow the advice of their advisory committees, but they often do. So while this is not a guaranteed approval, it is close to one.
What could happen once Epidiolex is on the market in the US?
This is where it gets interesting…
Off-Label Use – Epilepsy
The use of CBD for the treatment of epilepsy is nothing new. Multiple (albeit small) clinical trials dating back to the 1970s have demonstrated CBD efficacy in epilepsy.
It is conceivable that progressive neurologists will try Epidiolex in their adult patients before moving on to VNS and surgical treatment methods.
Effect on Non-Prescription Market
The use of CBD in neuropsychiatric disorders, such as anxiety, schizophrenia, and others has been postulated, and in some cases tried. Robust clinical trials are obviously lacking. However, there is no shortage of readily-available CBD-containing products, mainly oil-based oral solutions.
The medical claims surrounding some of these products range from the borderline legitimate to the comical.
The current size of the US market for non-prescription, medical CBD products is likely small, given the limited number of states which allow the sale of these products. However, the news of an FDA approval will likely drive interest in non-prescription CBD products, as well as THC:CBD blends.
Opioids Under Threat?
It is generally understood that CBD alone is not effective for the treatment of pain; some blend of THC and CBD is necessary for cannabis to have any chance of controlling pain. Thus, Epidiolex will not be prescribed off label for pain (at least it shouldn’t).
However, it is increasingly clear that the opioid market is under threat, and increased understanding of how CBD and THC work could enhance this threat. Pharma is taking note, as evidenced by the recent alliance between cannabis-grower Tilray and Sandoz Canada.
Having an FDA approved CBD product on the market, coupled with the efforts by different states to decriminalize and/or legalize medical cannabis for legitimate medical uses may result in the FDA finally making regulatory changes which will increase use of cannabis for legitimate medical purposes.
The Canadian regulatory model is an excellent framework for the FDA. And support for cannabis legalization in the US is quite strong, both from potential users/customers and government officials looking for incremental sales taxes.
So Where Is This All Headed?
We have seen market segments disrupted by completely new therapies, i.e., anti-TNF in psoriasis, HAART, etc. So having a new treatment modality enter and disrupt a therapeutic category is nothing new.
However, the confluence of an FDA approved product, popular sentiment towards legalization, and a flowering market in some large US states (and a number of other countries) suggests that a number of therapeutic areas may be significantly disrupted, even in the absence of FDA approved products.
This last point is key. Many patients living in California, Colorado, Canada, Germany, and other places who are suffering from chronic pain, Crohn’s Disease, or epilepsy are already trying and using a variety of cannabis-based products to manage disease.
These markets (and possibly others) are already well-primed for progressive pharmaceutical and non-pharmaceutical companies to step in and provide legitimate therapeutic options for thousands of patients globally.
Whether the industry establishment (corporate and regulatory) will aid or hamper this progress remains an open question.