Photo: The drug is a purified form of a chemical ingredient found in the cannabis plant. (ABC News: Jerry Rickard)
US health regulators have approved the first prescription drug made from marijuana, a milestone that could spur more research into a drug that remains illegal under federal law, despite growing legalisation for recreational and medical use.
The Food and Drug Administration (FDA) approved the medication, called Epidiolex, to treat two rare forms of epilepsy that begin in childhood. But it is not quite medical marijuana.
The strawberry-flavoured syrup is a purified form of a chemical ingredient found in the cannabis plant — but not the one that gets users high. It is not yet clear why the ingredient, called cannabidiol, or CBD, reduces seizures in some people with epilepsy.
British drugmaker GW Pharmaceuticals studied the drug in more than 500 children and adults with hard-to-treat seizures, overcoming numerous legal hurdles that have long stymied research into cannabis.
FDA officials said the drug reduced seizures when combined with older epilepsy drugs.
The FDA has previously approved synthetic versions of another cannabis ingredient for medical use, including severe weight loss in patients with HIV.
Epidiolex is essentially a pharmaceutical-grade version of CBD oil, which some parents already use to treat children with epilepsy. CBD is one of more than 100 chemicals found in marijuana. But it does not contain THC, the ingredient that gives marijuana its mind-altering effect.
Physicians said it was important to have a consistent, government-regulated version.
"I'm really happy we have a product that will be much cleaner and one that I know what it is," said Dr Ellaine Wirrell, director of the Mayo Clinic's program for childhood epilepsy.
"In the artisanal products there's often a huge variation in doses from bottle to bottle depending on where you get it."
Side-effects with the drug include diarrhea, vomiting, fatigue and sleep problems.
No immediate price for drug
Several years ago, Allison Hendershot considered relocating her family to Colorado, one of the first states to legalise marijuana and home to a large network of CBD producers and providers.
Her 13-year-old daughter, Molly, has suffered from severe seizures since she was four months old. But then Ms Hendershot learned about a trial of Epidiolex at New York University.
"I preferred this to some of those other options because it's is a commercial product that has gone through rigorous testing," Ms Hendershot said.
Since receiving Epidiolex, Ms Hendershot said her daughter had been able to concentrate more and had had fewer "drop" seizures — in which her entire body goes limp and collapses.
CBD oil is currently sold online and in specialty shops across the US, although its legal status remains murky. Most producers say their oil is made from hemp, a plant in the cannabis family that contains little THC and can be legally farmed in a number of states for clothing, food and other uses.
Charitable group Realm of Caring, affiliated with one of the US's largest CBD companies, estimated the typical family using CBD to treat childhood epilepsy spent about $US1,800 per year on the substance.
A GW Pharmaceuticals spokeswoman said the company would not immediately announce a price for the drug, which it expected to launch later this year.
Wall Street analysts previously predicted it could cost $US25,000 per year, with annual sales eventually reaching $US1 billion.
The FDA approval for Epidiolex is technically limited to patients with Dravet and Lennox-Gastaut syndromes, two rare forms of epilepsy for which there are few treatments. But doctors will have the option to prescribe it for other uses.
The new medication enters an increasingly complicated legal environment for marijuana.
Nine states and the District of Columbia have legalised marijuana for recreational use. Another 20 states allow medical marijuana, but the US Government continues to classify it as a controlled substance with no medical use, in the same category as heroin and LSD.
Originally published here: http://www.abc.net.au/news/2018-06-26/epilepsy-drug-made-from-marijuana-approved/9909378