First Marijuana-derived Drug Receives Boost from FDA

factotumk (32)in #cannabis • 6 years ago

Image source: Fortune

Boost from the FDA

In a FDA briefing published Tuesday, an experimental marijuana-derived drug received a favorable review from FDA scientists, bolstering hopes that it could become the first approved drug in the US made purely from marijuana.

Manufactured by UK-based GW Pharmaceuticals and marketed under the name Epidiolex, the drug would be the first approved drug of its kind to employ cannabidiol (CBD) as its active ingredient.

GW is seeking to sell the drug, a strawberry-flavored oral solution, to treat seizures associated with two forms of rare, early-onset epilepsy: Lennox-Gastaut (LGS) and Dravet (DS) syndromes in patients at least 2 years of age. These syndromes are resistant to many treatments, as evidenced by fact that 20 percent of children with DS die prior to adulthood.

FDA scientists released the following conclusions regarding Epidiolex's efficacy and safety:

"Clinically meaningful and statistically significant reductions in seizure frequency were demonstrated in three adequate and well-controlled trials in LGS and DS. The results from these three studies provide substantial evidence of the effectiveness of CBD for the treatment of seizures associated with LGS and DS. In general, the risks associated with CBD treatment appear acceptable, particularly given the findings of clinical efficacy in LGS and DS, which are serious, debilitating, and life-threatening disorders. Although the risk of liver injury has the potential to be serious, the observed risk can be appropriately managed with inclusion of relevant language in labeling, education of prescribers regarding the risk of transaminase elevation and need for monitoring of liver enzyme levels, and further characterization of the risk in the post-market setting. Although the review is still ongoing, the risk-benefit profile established by the data in the application appears to support approval of cannabidiol for the treatment of seizures associated with LGS and DS.”
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Scientists did note risk of liver injury, but contend physicians can mitigate liver risks by monitoring enzyme levels.

The FDA is expected to make an approval decision by June 27 and will consider the results from the briefing posted on Tuesday in addition to comments provided by external advisors with whom the FDA meets this Thursday. GW Pharmaceuticals had previously earned a protracted approval process after displaying that Epidiolex addresses a critical need.

Epidiolex: Hope for a Cure

Produced from a plant cultivated to yield a high CBD content, Epidiolex contains less than 0.1 percent of tetrahydrocannabinol (THC), the cannabinoid found in marijuana that is known to produce psychotropic effects. Thus, although cannabidiol is a cannabinoid like THC, it does not have the potential to create the euphoric high often associated with marijuana.

Some wondered if the fact that Epidiolex is marijuana-derived would hinder the drug’s approval. However, the FDA did not communicate any concerns, stating that “CBD has a negligible abuse potential.”

The FDA focused on two large clinical trials in its briefing. One trial analyzed the effects of liquid cannabidiol on 225 children with LGS. The subjects that were part of a group administered a high dose of the drug experienced their seizure occurrence drop by 42% (vs. a 17% reduction experienced in the placebo group). A second trial, published May 2017 in the New England Journal of Medicine, administered cannabidiol to 120 children with Dravet syndrome – 43% of participants given the drug saw their seizures reduced by 50% while the placebo group saw minimal improvement.

CBD: Schedule 1 Drug

Over a year ago, the DEA clarified that CBD, as a marijuana extract, is currently categorized as a Schedule 1 drug, meaning that it is federally illegal and has no accepted medical value. This is an interesting assertion considering the preponderance of recent medical studies that suggest many potential benefits for the cannabinoid:

Pain relief and anti-inflammatory properties
Smoking cessation and drug withdrawal
Epilepsy and mental health disorders
Anti-cancer agent
Reduction in anxiety disorders
Type 1 diabetes
Acne
Alzheimer’s disease

Some have even found CBD to be effective in boosting focus and productivity and mitigating stress associated with social anxiety.

Implications on future cannabis-derived drugs

If approved in June, Epidiolex would only be prescribed for DS and LGS patients. However, medical professionals would have the option to prescribe it “off-label” for other conditions. This could have significant implications in the US, where over 4.3 million individuals are affected by epilepsy. GW Pharmaceuticals has mentioned it is in the process of exploring treatments for other forms of epilepsy as well.

The FDA has previously approved drugs manufactured from synthethic cannabinoids including dronabinol, which is often marketed under Marinol or Syndros. These drugs have been proven to mitigate the loss of appetite associated with AIDS and nausea caused by chemotherapy.

May be unavailable in Colorado

In Colorado, it is unlawful for pharmacies to sell cannabis-derived products, while similarly, dispensaries are unable to sell FDA-approved medicines. That means children like Vienne Weinert Wood, who suffers from epilepsy despite trying seven different drugs, a special diet, and a brain pacemaker, may not have access to the drug. According to her mother , “The way it is right now in Colorado, I can get dog biscuits with CBD but I can’t get an FDA approved cannabis-based drug.” Representatives Lois Landraf and Janet Buckner are currently working to change that, sponsoring a bill authorizing pharmacies to sell Epidiolex.

What do you think?

Marijuana is currently a Schedule 1 drug, implying there are no medical benefits associated with the plant. Attorney General Jefferson Sessions has even worked to suppress medical marijuana research. However, many recent studies refute this perspective and suggest marijuana may offer a litany of medical benefits.

What do you think about the dichotomy between marijuana's status as a federally illegal, Schedule 1 drug and recent medical research?

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