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(CNN) – Epidiolex, the first cannabis-based drug approved by the US Food and Drug Administration, is now available on prescription in all 50 states. The twice-daily oral solution is approved for use in patients aged 2 years and older to treat two types of seizure syndrome: Dravet's syndrome, a rare genetic disorder of the brain that appears in the first year of life, and Lennox-Gastaut syndrome, one of epilepsy with several types of seizures that starts in early childhood, usually between 3 and 5 years of age.
"Because these patients have always reacted poorly to available medications, there is an urgent need for new treatments to reduce the frequency and impact of seizures," said Justin Gover, CEO of GW Pharmaceuticals, the manufacturer of Epidiolex, a written declaration. "We are committed to ensuring that these patients have access to this new cannabinoid-based drug that has been extensively studied in clinical trials and has been manufactured to ensure its quality. and uniformity and who is eligible to be covered by insurance for the appropriate patients.
Epidiolex was recommended for approval by an advisory committee in April and approved by the FDA in June. In September, the US Department of Justice and the Drug Enforcement Administration listed Epidiolex in the list of Schedule V substances, eliminating the last hurdle to its legal prescription by doctors in the United States. (Marijuana and CBD remain substances in Table I.)
"Adequate and well-controlled clinical studies have approved the approval of Epidiolex. Prescribers can therefore trust the consistent strength of the drug and its consistent delivery that supports the appropriate dosage required to treat patients with these complex and severe epileptic syndromes, "said Dr. Scott Gottlieb, FDA Commissioner , a written statement. "The FDA will continue to support rigorous scientific research on the potential medical uses of marijuana products and will be ready to work with product developers who want to provide patients with safe, effective and high quality products."
Dr. Orrin Devinsky, director of the Comprehensive Epilepsy Center at NYU Langone Health, has been the principal investigator of two of Epidiolex's three Phase III clinical trials.
"In these syndromes, when [Epidiolex] has been added to three more seizures [medications]on average, he reduced seizures – or "gout attacks" – from about 25% to 28% compared to a placebo, "said Devinsky. "So I think it's very important to recognize … that it's clear, it's effective, and it was statistically significant in the three big studies we did, but at the same time, the effect was modest .
"Some people have seen dramatic improvements. Many had a modest improvement, and some had no improvement. So it's not a miracle drug. It's an effective drug, and I think its side-effect profile is pretty good compared to other seizure medications we have, but it's not a miracle cure, "Devinsky said.
According to GW Pharmaceuticals, the most common side effects of Epidiolex are drowsiness, loss of appetite, diarrhea and increased liver enzymes.
Shauna Garris, a pharmacist, clinical pharmacy specialist and assistant professor at the Eshelman School of Pharmacy at the University of North Carolina, said the drug was effective and that it worked between "enough" and "very well". She did not use Epidiolex in her own clinical practice and did not participate in the development of the drug, but added that she was not sure she would live up to "everything." the hype "that surrounded him.
She said, however, that the FDA's approval of Epidiolex signals "a validation of the science of cannabinoid-based drugs."
GW Pharmaceuticals reports that the average price of Epidiolex is $ 32,500 per year. The company expects the drug to be covered by most insurance plans and claims that "this price matches that of other brand-name antiepileptic drugs approved by the FDA, such as Banzel." , and the access of eligible patients should be limited. similar to such DAEs for similar indications for LGS (eg, Onfi and Banzel). "
It is also launching a patient support program "to help patients who have been prescribed Epidiolex to access treatment. The program provides education and patient / caregiver-focused resources to help reduce direct costs or provide a no-cost product to eligible patients. "
Although Epidiolex is only approved for the treatment of two rare convulsive disorders, doctors can now prescribe the drug "non-compliant" under other conditions. According to the US Department of Health and Social Services, this is both legal and common; One in five drugs is prescribed for use not indicated on the label.
"The unauthorized use of CBD and Epidiolex will be a fascinating subject to look at over the next few years," Devinsky said. "Many, many patients would like to access it without meeting the criteria, and I believe that many doctors will try to prescribe it, as well as for some people who, I think, have tried many drugs and for whom could be beneficial . "
He added, "I think that doctors are always trying to make good benefit / toxicity decisions, and that CBD is a pretty attractive compound. The question is, what will insurance companies do?
(The-CNN-Wire ™ and © 2018, Cable News Network, Inc., a Time Warner Company, All Rights Reserved.)
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