Introduction of First Medication with Marijuana-Derived Component in US

Key Takeaways:

• The first medication incorporating a component from the cannabis plant, Epidiolex, has been approved by the FDA and released in the US. This medication is specifically designed for patients aged two years and above experiencing Dravet syndrome and Lennox-Gastaut syndrome.
• The FDA approval comes in light of thorough clinical studies that validate the consistent strength and delivery of the drug, instilling confidence in prescribers.
• In clinical trials, it was found that even lower dosages of Epidiolex, specifically a 10mg daily dosage, could effectively reduce seizures with fewer side effects, making it a preferred choice among parents.
• The study also assured that Epidiolex is a safe treatment with minimal patient dropouts owing to side effects, although potential side effects such as poor appetite, lethargy, sleepiness, can occur.
• Further studies are needed to understand CBD’s efficacy in treating different types of epilepsy, with a call for larger research initiatives in the field of focal epilepsy.

The first medication containing a component sourced from the cannabis plant recently debuted in the US following its approval by the Food and Drug Administration (FDA). The medication, Epidiolex, is a CBD (cannabidiol) oral solution composed twice daily and produced by GW Pharmaceuticals, based in the United Kingdom.

Therapeutic Potential and FDA Approval

The drug got approved for patients aged two years and above suffering from two types of epileptic syndromes, namely Dravet syndrome, and Lennox-Gastaut syndrome. According to Justin Gover, GW Pharmaceuticals’ CEO, this approval is significant as these epileptic conditions have historically demonstrated little response to established seizure medication. Thus, there has been a desperate need for novel therapies that aim to minimize the frequency and impact of seizures.

In a June statement, FDA Commissioner, Dr. Scott Gottlieb reiterated regulatory approval and oversight’s importance in ensuring patient safety. This green light was partly due to adequate and well-planned clinical studies that backed the approval, which gives prescribers confidence in the drug’s consistent strength and delivery.

Efficacy of Low Dosages

While CBD has enjoyed popularity across the US in recent years for dealing with various conditions, including pain, the evidence for its effectiveness is substantial only in treating epilepsy. Epidiolex grabbed headlines in May after a clinical trial demonstrated that even low dosages of the drug could aid patients suffering from epilepsy.

During the trial, patients using pharmaceutical-grade cannabidiol in a 10mg daily dosage experienced nearly as much reduction in seizures as those using 20mg, but with lower side effects, according to the study’s lead researcher, Dr. Orrin Devinsky, who is also Director of NYU Langone’s Comprehensive Epilepsy Center in New York City.

Role of Dosage

While the 20mg dosage was slightly more effective, it wasn’t the preferred choice among parents. These patients did not suffer as many side effects as CBD can cause, such as decreased appetite, tiredness, diarrhea, and possible signs of liver damage, according to Devinsky.

Compellingly, the study also demonstrated that Epidiolex is a safe treatment, with only seven patients dropping out due to side effects. Notably, this included six from the 20mg group and only one from the 10mg cohort. Despite this, the FDA, in its approval notice, highlighted that side effects could occur, such as poor appetite, lethargy, sleepiness, infections, and others.

Utility in Severe Cases

Dr. Angel Hernandez, of the Helen DeVos Children’s Hospital in Grand Rapids, asserts that these findings validate that pharmaceutical-grade CBD can suppress seizures and “increases our options to treat many of these patients with very, very difficult-to-control epilepsies.”

While the current results are promising, both Hernandez and Devinsky agreed that there is a need for more studies, especially concerning CBD’s performance in treating different epilepsy forms. In particular, Devinsky called for a larger study in focal epilepsy.

The promising findings of the Epidiolex study were published online on May 17 in the New England Journal of Medicine.

Additional Information

Find more about medical marijuana and epilepsy from the Epilepsy Foundation.