FDA Approves First Marijuana Based Pharmaceutical

The U.S. Food and Drug Administration has for the first time Monday, approved a marijuana based pharmaceutical drug, Epidiolex, for treatment of two extremely severe and treatment-resistant forms of epilepsy (Lennox-Gastaut syndrome and Dravet syndrome), that tragically affect infants and young children.

The treatment is an oral solution derived from active ingredients in the marijuana plant, but not those compounds that produce the characteristic “high” associated with marijuana and sought out by recreational consumers for the sense of relief and relaxation it brings, enhanced sensory experiences, and creative insight.

That euphoric state is caused by a cannabinoid (a class of naturally occurring macro-molecules that dial the levels of various neurotransmitters up and down in animal brains, altering their mode of consciousness) called delta-9-tetrahydrocannabinol or THC.

The active ingredient in Epidiolex, however, is cannabidiol or CBD, another compound found in marijuana that doesn’t have the intoxicating effects of THC, but has emerged through medical research as a miracle treatment for infants and small children suffering from severe forms of epilepsy that are profoundly devastating to their quality of life and healthy development because of the severity and frequency of the seizures, and because of their resistance to typical, non-marijuana based pharmaceutical anti-seizure medications. These desperate children and their families have been given a new hope for a better life.

As the FDA summarizes in a press release:

“Dravet syndrome is a rare genetic condition that appears during the first year of life with frequent fever-related seizures (febrile seizures). Later, other types of seizures typically arise, including myoclonic seizures (involuntary muscle spasms). Additionally, status epilepticus, a potentially life-threatening state of continuous seizure activity requiring emergency medical care, may occur. Children with Dravet syndrome typically experience poor development of language and motor skills, hyperactivity and difficulty relating to others.

Lennox-Gastaut syndrome begins in childhood. It is characterized by multiple types of seizures. People with Lennox-Gastaut syndrome begin having frequent seizures in early childhood, usually between ages 3 and 5. More than three-quarters of affected individuals have tonic seizures, which cause the muscles to contract uncontrollably. Almost all children with Lennox-Gastaut syndrome develop learning problems and intellectual disability. Many also have delayed development of motor skills such as sitting and crawling. Most people with Lennox-Gastaut syndrome require help with usual activities of daily living.”

Justin Gover, CEO of GW Pharmaceuticals, called the FDA approval “a historic milestone.”

He added that the drug offers families “the first and only FDA-approved cannabidiol medicine to treat two severe, childhood-onset epilepsies. These patients deserve and will soon have access to a cannabinoid medicine that has been thoroughly studied in clinical trials, manufactured to assure quality and consistency, and available by prescription under a physician’s care.”

This approval is not only a medical milestone promising a better life for suffering children and their families, but a legal one that portends a reorganization of U.S. drug policy on a federal level, because this first-ever FDA approval of a marijuana-based pharmaceutical now precludes marijuana from the legal definition of a Schedule I Substance under the U.S. Controlled Substances Act of 1970.

Under the scheduling schema of the CSA, which categorizes scheduled (illicit) drugs into five categories or schedules, Schedule I substances are considered the most harmful and penalties for manufacturing, selling, and possessing these drugs are the most severe.

But to be included under Schedule I under the CSA:

(A) The drug or other substance has a high potential for abuse.
(B) The drug or other substance has no currently accepted medical use in treatment in the United States.
(C) There is a lack of accepted safety for use of the drug or other substance under medical supervision.

If we think of that legal definition of a Schedule I substance as a three-legged stool, the FDA’s approval of a marijuana-based pharmaceutical this week just knocked out two of the three legs.

Now it will be up to the Drug Enforcement Administration to reclassify marijuana, although Congress has its eye on various statutory reforms of federal marijuana policy as well.