A ground-breaking cannabidiol oral solution (Epidiolex, GW Pharmaceuticals) is now available by prescription in the United States to treat seizures associated with Dravet syndrome or Lennox-Gastaut syndrome (LGS) in patients aged 2 years or older.1
Approved by the FDA in June, Epidiolex is the first prescription pharmaceutical formulation of highly purified, plant-derived cannabidiol (CBD), a cannabinoid lacking the high associated with marijuana, and the first in a new category of anti-epileptic drugs.1,2
Epidiolex provides a new treatment option for patients with 2 of the most difficult-to-treat forms of childhood-onset epilepsy, according to Justin Gover, chief executive officer of GW Pharmaceuticals.1
“Because these patients have historically not responded well to available seizure medications, there has been a dire need for new therapies that aim to reduce the frequency and impact of seizures,” he said in a statement.1 “We are committed to ensuring that these patients can access this novel cannabinoid medicine that has been thoroughly studied in clinical trials, manufactured to assure quality and consistency, and is eligible to be covered by insurance for appropriate patients.”
In a statement to Pharmacy Times, the American Epilepsy Society (AES) voiced support for the FDA's approval of Epidiolex, as a treatment option for these 2 rare forms of epilepsy. However, the society also encouraged patients to be cautious with this new medication.
“The AES cautions that although prescription cannabidiol medication is made from the cannabis plant, it is a highly purified and carefully manufactured oral formulation and is not the same as medical marijuana, which may also contain THC (which causes a high), pesticides, and other dangerous impurities,” AES officials said in the statement. “Further, while trials have shown that pharmaceutical-grade CBD product (which cannot be obtained from a marijuana dispensary) can be effective, it’s not a silver bullet and also has more side effects and can interact with other epilepsy medications.”
Epidiolex’s effectiveness was studied in 3 randomized, double-blind, placebo-controlled clinical trials involving 516 patients with either Dravet syndrome or LGS. Epidiolex, taken along with other medications, was shown to be effective in reducing the frequency of seizures compared with the placebo.2
The most common adverse effects that occurred in Epidiolex-treated patients in the clinical trials were sleepiness, sedation and lethargy; elevated liver enzymes; decreased appetite; diarrhea; rash; fatigue, malaise and weakness; insomnia, sleep disorder and poor quality sleep; and infections.2
In launching the new drug treatment, GW Pharmaceuticals and its United States subsidiary Greenwich Biosciences have also introduced a comprehensive patient support program, Epidiolex Engage, that aims to help patients who have been prescribed Epidiolex gain access to therapy.
The program offers caregiver- and patient-focused education and resources to help lower out-of-pocket costs or provide product at no cost for eligible patients, according to the company.1
In a statement, Christina SanInocencio, executive director of the Lennox-Gastaut Syndrome Foundation, said that LGS is a devastating form of epilepsy.3
"Epidiolex is a much-needed new treatment option for patients with LGS, a rare and severe form of childhood-onset epilepsy that typically persists into adulthood,” she said in a statement.1 “Despite the use of multiple epilepsy treatments, the majority of LGS patients continue to have life-long, debilitating seizures, and our community welcomes the availability of a new, first-in-class treatment option.”
Mary Anne Meskis, executive director of the Dravet Syndrome Foundation, also expressed support for the new treatment in prepared statements.
“While each of our disorders are different in terms of etiology, both of our communities suffer from intractable seizures, an increased risk of mortality, and many devastating co-morbid conditions that significantly affect quality of life,” she said.3
The Dravet syndrome community has long desired a medication specifically approved for the treatment of seizures associated with the condition, Meskis said.
"The availability of Epidiolex is an important milestone for patients and caregivers whose lives are significantly impacted by this catastrophic, lifelong form of epilepsy,” she said.1