FDA Approves New Indication For Drug Containing An Active Ingredient Derived From Cannabis To Treat Seizures In Rare Genetic Disease

DISTRICT OF COLUMBIA: Last month, the U.S. Food and Drug Administration approved Epidiolex (cannabidiol) [CBD] oral solution for the treatment of seizures associated with tuberous sclerosis complex (TSC) in patients one year of age and older. Epidiolex was previously approved for the treatment of seizures associated with two rare and severe forms of epilepsy, Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS). This is the only FDA-approved drug that contains a purified drug substance derived from cannabis. It is also the second FDA approval of a drug for the treatment of seizures associated with TSC.

CBD is a chemical component of the Cannabis sativa plant. However, CBD does not cause intoxication or euphoria (the “high”) that comes from tetrahydrocannabinol (THC). It is THC (and not CBD) that is the primary psychoactive component of cannabis.

“The FDA continues to believe the drug approval process represents the best way to make new medicines, including any drugs derived from cannabis, available to patients in need of appropriate medical therapy such as the treatment of seizures associated with these rare conditions. This paradigm ensures new therapies are safe, effective, and manufactured to a high quality that provides uniform and reliable dosing for patients,” said Douglas Throckmorton, M.D., deputy center director for regulatory programs in the FDA’s Center for Drug Evaluation and Research. “The agency is committed to supporting rigorous scientific research on the potential medical uses of cannabis-derived products and working with product developers who are interested in bringing patients safe and effective, high quality products.”

TSC is a rare genetic disease that causes non-cancerous (benign) tumors to grow in the brain and other parts of the body like the eyes, heart, kidneys, lungs, and skin. TSC usually affects the central nervous system and can result in a combination of symptoms including seizures, developmental delay, and behavioral problems, although the signs and symptoms of the condition, as well as the severity of symptoms, vary widely. TSC affects about 1 in 6,000 people.

Epidiolex’s effectiveness for the treatment of seizures associated with TSC was established in a randomized, double-blind, placebo-controlled trial where 148 patients out of a total of 224 in the study received Epidiolex. The study measured the change from baseline in seizure frequency. In the study, patients treated with Epidiolex had a significantly greater reduction in the frequency of seizures during the treatment period than patients who received placebo (inactive treatment). This effect was seen within eight weeks and remained consistent throughout the 16-week treatment period.

The most common side effects that occurred in Epidiolex-treated patients with TSC in the clinical trial were: diarrhea, elevated liver enzymes, decreased appetite, sleepiness, fever, and vomiting. Additional side effects for patients with LGS, DS, or TSC include: liver injury, decreased weight, anemia, and increased creatinine.

Epidiolex must be dispensed with a patient Medication Guide that describes important information about the drug’s uses and risks. As is true for all drugs that currently treat epilepsy, including Epidiolex, the most serious risks may include an increase in suicidal thoughts and behavior, or thoughts of self-harm. Patients, their caregivers, and their families should be advised to monitor for any unusual changes in mood or behavior, such as worsening depression, suicidal thoughts or behavior. Patients, caregivers, and families should report behaviors of concern immediately to healthcare providers. Epidiolex also caused liver injury in some patients. Most cases were generally mild, but a risk of rare, but more severe liver injury exists. More severe liver injury can cause nausea, vomiting, abdominal pain, fatigue, anorexia, jaundice, and/or dark urine.

The FDA granted Priority Review designation for this application. The approval of Epidiolex was granted to Greenwich Biosciences Inc., of Carlsbad, California.

FDA Approves First Cannabis-Based Epilepsy Drug

MARYLAND: The U.S. Food and Drug Administration has approved Epidiolex (cannabidiol) [CBD] oral solution for the treatment of seizures associated with two rare and severe forms of epilepsy, Lennox-Gastaut syndrome and Dravet syndrome, in patients two years of age and older. This is the first FDA-approved drug that contains a purified drug substance derived from marijuana. It is also the first FDA approval of a drug for the treatment of patients with Dravet syndrome.

CBD is a chemical component of the Cannabis sativa plant, more commonly known as marijuana. However, CBD does not cause intoxication or euphoria (the “high”) that comes from tetrahydrocannabinol (THC). It is THC (and not CBD) that is the primary psychoactive component of marijuana.

“This approval serves as a reminder that advancing sound development programs that properly evaluate active ingredients contained in marijuana can lead to important medical therapies. And, the FDA is committed to this kind of careful scientific research and drug development,” said FDA Commissioner Scott Gottlieb, M.D. “Controlled clinical trials testing the safety and efficacy of a drug, along with careful review through the FDA’s drug approval process, is the most appropriate way to bring marijuana-derived treatments to patients. Because of the adequate and well-controlled clinical studies that supported this approval, prescribers can have confidence in the drug’s uniform strength and consistent delivery that support appropriate dosing needed for treating patients with these complex and serious epilepsy syndromes. We’ll continue to support rigorous scientific research on the potential medical uses of marijuana-derived products and work with product developers who are interested in bringing patients safe and effective, high quality products. But, at the same time, we are prepared to take action when we see the illegal marketing of CBD-containing products with serious, unproven medical claims. Marketing unapproved products, with uncertain dosages and formulations can keep patients from accessing appropriate, recognized therapies to treat serious and even fatal diseases.”

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.

“The difficult-to-control seizures that patients with Dravet syndrome and Lennox-Gastaut syndrome experience have a profound impact on these patients’ quality of life,” said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “In addition to another important treatment option for Lennox-Gastaut patients, this first-ever approval of a drug specifically for Dravet patients will provide a significant and needed improvement in the therapeutic approach to caring for people with this condition.”

Growing Hope: The Fight For Medical Marijuana For Epileptic Kids

COLORADO:  January 22nd 2015 was a brisk, cold day. All the trees in Richmond, Virginia were bare from a harsh winter. As the chill blew up the hill in the city’s historic Shockoe Slip district, several families began to emerge from a hotel in the shadow of the State Capitol.

Lisa Smith was there with her 14-year-old daughter, Haley. Beth and Patrick Collins were there too, accompanied by their 15-year-old daughter Jennifer. Later, they would be joined by Teresa Elder, her daughter Ashley, and Teresa’s 22-year-old son, Tommy.

Lisa Smith and her daughter Haley with Virginia House Speaker James Howell, Republican.

Haley, Jennifer and Tommy are desperately ill with extreme forms of epilepsy. They have illnesses so insidious they have stymied a virtual army of doctors and specialists. But these families believe that oils made from a cannabis plant might help their children where they say all other anti-epilepsy drugs have failed.

Colorado Springs Student Suspended For Bringing Medical Marijuana To School

COLORADO:  A disabled Sand Creek High School student was suspended Monday after school officials discovered medical marijuana in his lunch box, according to Gazette news partner KKTV.

Jackson Stormes, 16, has constant seizures because of Dravet Syndrome, which has left him severely disabled. His mother, Jennie Stormes, says cannabis is the only medicine that has stemmed the seizures. Instead of seizing for 30 to 60 minutes at a time, the episodes last only two to three minutes.

The family moved from New Jersey to get better access to medical marijuana. Jennie Stormes said she accidentally put yogurt mixed with cannabis pills in Jackson’s lunch on Monday, and she got a call from the school around lunch that he had been suspended for bringing a controlled substance to school.

WellSpan Prepares To Launch Region’s First Medical Marijuana Study

PENNSYLVANIA:  At WellSpan Health, pediatric neurologist Dr. Todd Barron said about 50 percent of his patients have epilepsy.

Medical marijuana isn’t a foreign topic, as anecdotal evidence suggests the CBD compound in the plant can help with seizures, he said.

With no definite answer — yet — Barron said WellSpan will be conducting two studies that will test CBD on epilepsy: one for children with Dravet syndrome and one for those with Lennox–Gastaut syndrome.

“We felt that this was the best way to help our patients, by looking at the drug in a safe fashion,” he said.

 

The Medical Marijuana Fight Hits Virginia

VIRGINIA: The debate continues about whether or not to legalize medical marijuana. Not just across the country, but in Virginia as well.

It’s an issue impacting many families, including one here in the Valley.

Two families, both with roots in Virginia, find themselves at the center of the debate. Fighting against lawmakers and government agencies to change the medicine their children receive in order to save their lives, each using a form of medical marijuana.

Moms’ Marijuana-For-Kids Campaign Seeks To Quiet Epilepsy

UTAH:  April Sintz is fighting to loosen marijuana laws for her 7-year-old epileptic son. She is one of hundreds of moms nationwide who have opened a new front in the drive to expand the drug’s legal use.

While supplying pot to a child is bound to raise eyebrows, Sintz said early evidence on the marijuana extract cannabidiol, also known as CBD, suggests it’s a potent anticonvulsant, with few dangerous side effects. That could help save the life of her son, Isaac, who has 30 seizures or so a day and suffers with kidney damage from his present treatments, she said.

“We’re probably going to lose our son to his kidneys or his seizures,” said Sintz, who lives in South Jordan, Utah, near Salt Lake City, and whose son had his first seizure at 6 months old. “We can’t find a medication to safely control those seizures, which is why we’re so excited for this oil.”

 

 

Six-Year-Old Medical Marijuana User Runs Afoul Of Health Canada Rules

CANADA:  Liam McKnight signed his medical marijuana licence when he was just five years old.

The boy from Constance Bay suffers from Dravet syndrome, a rare and severe form of epilepsy. It can cause nearly constant seizures that last three to four minutes each. His condition led him to miss time during kindergarten.

Liam had 67 seizures the day before starting cannabis oil treatment. The first 10 days he used cannabis oil, he was seizure-free, his mother says.

“He had new words,” said Liam’s mother, Mandy. “He was horseback riding. He was in a boat, he went tubing. He was so happy. We had a little glimpse of what life could be like.”

Even though Liam is licensed to use medical marijuana, taking it in extracted oil form violates Health Canada’s new Marihuana for Medical Purposes Regulations, which came into effect April 1.

 

Ohio Needs Heroes

OHIO: Paige needs a hero.  Ohio needs a lot of heroes.  The little girl in this poignant video is but one of many thousands of Ohioans who are suffering due to a lack of safe and legal access to therapeutic cannabis.

We have many families in Ohio with kids who suffer from Dravet Syndrome and epilepsy who could surely benefit from this amazing plant.  Presently Ohio families with children like Paige, have to make tough choices: move for treatment and leave behind their families, careers and homes, or stay put and endure the barrage of seizures and permanent brain damage that come with these debilitating diseases. [Read more…]

Can Marijuana Make John Hanger Governor?

PENNSYLVANIA:  John Hanger might not be the first guy you’d think of to be the “pot candidate” in this year’s Pennsylvania gubernatorial campaign: He’s a Penn Law grad who has held several reasonably high-profile state posts during his career.

Still, it’s Hanger’s aggressive pro-legalization stance that has come to define his candidacy. He both embraces that distinction — believing it might get him the numbers to win the Democratic primary — and eschews it, saying that he’s got plans and proposals to address a host of issues afflicting the state.

Talk to him, and you might find yourself suspecting Hanger could benefit from a toke or two. He can be frenetic in conversation, packing thousands of words and many ideas into a compact space, as though he were racing to beat an invisible timer. And that’s when he’s focused on the conversation — he interrupted his phone call with Philly Mag last week to do campaign business, offering buttons and bumper stickers to supporters. “We’re going to knock those elites out of office,” he promised. [Read more…]