FDA panel recommends Cannabis derived drug for Epilepsy
The federal panel recommended the approval of the first prescription cannabidiol medicine which may be used to treat rare and severe forms of epilepsy.
The advisory committee to the Food and Drug Administration unanimously voted to recommend approval of the drug, known as Epidiolex. This would be the first prescription medicine derived from plant-based cannabidiol(CBD)
Cannabidiol — CBD for short — is a cannabis derivative and the approval would be limited to treating seizures caused by Lennox-Gastaut syndrome and Dravet syndrome in patients aged 2 and older.
The Centers for Disease Control and Prevention reported that epilepsy is becoming more commonly reported in the U.S. Three million adults report they have epilepsy, up from 2.3 million in 2010
“Epidiolex represents hope for the many individuals living with intractable seizures and rare epilepsies, who every day face incredible challenges and disabling seizures, and live with the continual risk of serious injury and death,” said Philip Gattone, president, and CEO of the Epilepsy Foundation.
The new experimental medication utilizes a chemical compound in cannabis that doesn’t get a user high, unlike tetrahydrocannabinol, or THC.
Following features make the product unique-
A British company, GW Pharmaceuticals, has gone through the testing necessary to be considered for FDA approval.
Its carefully formulated syrup-like oral medicine will have to be made under guidelines, called good manufacturing practices so that buyers can count on getting what is supposed to be in the bottle.
"It's incredibly important as a physician prescribing to patients that we know what we're giving them and we know we give them one month will be the same that we give them three or six months later," said Dr. Orrin Devinsky, director of NYU Langone Epilepsy Center.
"It's very important to highlight that the drug used in this study, cannabidiol, was derived from cannabis plants, and purified to 99 percent purity," Devinsky told NBC News.
"This is not something you can get from a dispensary today in the United States. It's not something that you can get by smoking marijuana. It is a very specific pharmaceutical grade product derived from marijuana, but quite different than what is available anywhere else right now in the United States."
“Both by Lennox-Gastaut syndrome and Dravet syndrome are rare, severe, refractory epilepsy syndromes with onset in early childhood,” the FDA said.
It’s not clear how or why CBD works, but studies show it does, the FDA said.
“The results from these three studies provide substantial evidence of the effectiveness of CBD for the treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome (LGS and DS),” the FDA said in briefing documents given to the advisory committee ahead of Thursday’s meeting.
“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.”
The panelists heard the case from several desperate parents, including Lisa Smith of White Stone, Virginia, whose teenaged daughter Haley was suffering from Dravet Syndrome and has undergone thousands of seizures.
“By the time she was 14, she had tried and failed 17 medications and combinations of them. The doctors said there was nothing left," Smith, an activist for the legalization of marijuana-based medicines, told the panel.
"So what do you do, when you have a 14-year-old, and there was nothing? They said they’d prescribe CBD only if they wouldn’t be arrested.”
Dravet syndrome is very rare and often deadly form of epilepsy caused by a genetic mutation. These kids have multiple, prolonged seizures that cause brain damage. Lennox-Gastaut syndrome is also rare, causes various types of seizures and can also cause severe cognitive impairment.
Devinsky exemplifies the work of CBD:
"There are some children I take care of who are literally wheelchair-bound, having hundreds of seizures a day on high doses of other medications and who are either sleeping or seizing most of their life," he said.
"Some of these children are now out of the wheelchair running around, kicking soccer balls, going to school -- able to enjoy life with their family. It has been an amazing transformation for some of these children."
Studies have shown that it can have some benefits in reducing anxiety, counteracting some types of nausea and that it may relieve some symptoms of glaucoma and multiple sclerosis.
And the FDA has approved several marijuana-based products, including the synthetic formulation Marinol, prescribed to treat severe appetite loss and nausea in conditions such as AIDS.
Separately, the Centers for Disease Control and Prevention reported Thursday that epilepsy is becoming more commonly reported in the U.S. Three million adults report they have epilepsy, up from 2.3 million in 2010
Doctors are interested in trying CBD to treat autism, anxiety, inflammatory and autoimmune disorders also.