Questions abound about availability, legality of new cannabis-derived epilepsy drug

 

The first-ever federal approval of a marijuana-derived drug, Epidiolex (cannabidiol), comes with a cloud of complications.

The drug, approved by the Food and Drug Administration in June to treat Lennox-Gastaut syndrome and Dravet syndrome in patients aged 2 years or older, is expected to be so expensive that those seeking it for off-label uses may join those who buy unregulated cannabis products on the open market. And legal complications could prevent some neurologists from prescribing Epidiolex at its planned fall launch.

Epidiolex is an oral solution of purified cannabidiol (CBD), a component of the marijuana plant that does not make people high.

According to the results of a phase 3 trial in patients with Lennox-Gastaut syndrome published earlier this year, median monthly drop seizures fell by 43.9% in a Epidiolex group compared with 21.8% in a placebo group (Lancet. 2018;391[10125]:1085-96). A 2017 study found that patients with Dravet syndrome who took the drug had fewer median convulsive seizures per month, dropping from 12.4 to 5.9, while there was barely a difference for the placebo group (N Engl J Med. 2017;376:2011-20). In both trials, patients received Epidiolex as an add-on treatment.

“There definitely are side effects such as sleepiness, abnormal liver function values, and other things that people will have to watch out for, such as interactions with other drugs,” Jacqueline A. French, MD, professor of neurology at New York University and chief scientific officer of the Epilepsy Foundation, said in an interview. “But it will definitely be a great benefit to some and a benefit to many.”

It’s not clear how the drug works, she said.

Greenwich Biosciences, the U.S. subsidiary of British drug maker GW Pharmaceuticals, plans to market Epidiolex this fall, spokesman Steve Schultz said in an interview. First, however, the U.S. Drug Enforcement Agency must reschedule the drug so it doesn’t fall under federal antimarijuana laws. It has 90 days after the FDA approval to do so, and its rescheduling is expected.

Then the focus will turn to the states, which have a crazy quilt of laws about marijuana and its medical use. “We’ve worked with the state legislators to enact laws or to modify laws to allow for our medicine to be made available,” Mr. Schultz said. “There may be one or two that we’re still working on.”

At issue: States and the District of Columbia have a wide variety of laws that appear to affect the sale of Epidiolex.

According to the website procon.org, 30 states and the District of Columbia allow medical marijuana, although the laws vary regarding what is allowed and do not address cannabis-derived pharmaceuticals.

Another 17 states have laws that address CBD specifically, often with language that allows its use to treat epilepsy in all or specific cases, according to procon.org.

The Kentucky law, for instance, only allows prescriptions by hospitals and clinics linked to public universities. A law in Missouri, according to procon.org, only allows the use of certain kinds of “cannabis oil” for intractable epilepsy and “requires a neurologist to determine that the patient did not respond to at least three treatment options to be eligible to use the marijuana extract.”

 

In Oklahoma, an anticannabis law excludes “from the definition of marijuana ‘any federal Food and Drug Administration–approved cannabidiol drug or substance.’ ”

North Carolina attorney Rod Kight, who represents businesses in the cannabis industry, predicts that “once the federal government [via the DEA] changes the law, the states will fall in line. The only thing that would prevent them would be a lack of understanding of what’s going on.”

Meanwhile, many sellers of CBD-based products continue to promote their use as treatments for epilepsy and many other conditions.

Legal exceptions made by states for FDA-approved CBD-based drugs could affect access to unapproved CBD-based products. It’s also possible that Epidiolex could hurt sellers of cannabis products in other ways. “They’ll now have to compete with a drug made to a specific strength and purity, and that’s probably going to have some impact on their business,” William J. McNichol, adjunct professor at Rutgers Law School, Camden, N.J., said in an interview. “If you have a choice, are you going to choose aspirin I made myself in my artisan aspirin factory that the FDA never saw? Or aspirin made by Bayer?”

But there’s a big obstacle to the widespread use of Epidiolex, especially for off-label uses for other types of epilepsy: It’s expected to cost $25,000 a year.

What’s next? For one, the FDA’s approval of Epidiolex seems likely to open the door for more cannabis-based medications. “The FDA is quite open to evaluating cannabinoid-based medicines as long as they go through their process,” said Mr. Schultz, the Greenwich Biosciences spokesman.

As for the legal front, Mr. Kight said potential changes in federal law could expand the legality of cannabis-based products by allowing them when they’re derived from “industrial hemp.” This could mean that patients with epilepsy will have more legal ways to buy CBD products.

Regardless of the legal situation, Mr. Kight said, “a physician can explain to patients across the board about how CBD might benefit them. The physician can say there are extract products that are out there and available over the counter.”

As for the maker of Epidiolex, GW Pharmaceuticals is recruiting for a phase 3 trial of a cannabinoid treatment for tuberous sclerosis complex, Mr. Schultz said. The results are due in the first half of 2019.

The company is also conducting cannabinoid research in the areas of autism and Rett syndrome, Mr. Schultz said, adding: “We also have done work in schizophrenia and in oncology and glioblastoma.”

Mr. Kight disclosed that he represents companies in the cannabis industry. Dr. French is president of the Epilepsy Study Consortium and disclosed working with multiple drug makers in the epilepsy field, including GW Pharmaceuticals. Dr. French receives fixed compensation by the consortium and is not paid by any pharmaceutical company. Mr. McNichol reported no relevant disclosures.

 

The first-ever federal approval of a marijuana-derived drug, Epidiolex (cannabidiol), comes with a cloud of complications.

The drug, approved by the Food and Drug Administration in June to treat Lennox-Gastaut syndrome and Dravet syndrome in patients aged 2 years or older, is expected to be so expensive that those seeking it for off-label uses may join those who buy unregulated cannabis products on the open market. And legal complications could prevent some neurologists from prescribing Epidiolex at its planned fall launch.

Epidiolex is an oral solution of purified cannabidiol (CBD), a component of the marijuana plant that does not make people high.

According to the results of a phase 3 trial in patients with Lennox-Gastaut syndrome published earlier this year, median monthly drop seizures fell by 43.9% in a Epidiolex group compared with 21.8% in a placebo group (Lancet. 2018;391[10125]:1085-96). A 2017 study found that patients with Dravet syndrome who took the drug had fewer median convulsive seizures per month, dropping from 12.4 to 5.9, while there was barely a difference for the placebo group (N Engl J Med. 2017;376:2011-20). In both trials, patients received Epidiolex as an add-on treatment.

“There definitely are side effects such as sleepiness, abnormal liver function values, and other things that people will have to watch out for, such as interactions with other drugs,” Jacqueline A. French, MD, professor of neurology at New York University and chief scientific officer of the Epilepsy Foundation, said in an interview. “But it will definitely be a great benefit to some and a benefit to many.”

It’s not clear how the drug works, she said.

Greenwich Biosciences, the U.S. subsidiary of British drug maker GW Pharmaceuticals, plans to market Epidiolex this fall, spokesman Steve Schultz said in an interview. First, however, the U.S. Drug Enforcement Agency must reschedule the drug so it doesn’t fall under federal antimarijuana laws. It has 90 days after the FDA approval to do so, and its rescheduling is expected.

Then the focus will turn to the states, which have a crazy quilt of laws about marijuana and its medical use. “We’ve worked with the state legislators to enact laws or to modify laws to allow for our medicine to be made available,” Mr. Schultz said. “There may be one or two that we’re still working on.”

At issue: States and the District of Columbia have a wide variety of laws that appear to affect the sale of Epidiolex.

According to the website procon.org, 30 states and the District of Columbia allow medical marijuana, although the laws vary regarding what is allowed and do not address cannabis-derived pharmaceuticals.

Another 17 states have laws that address CBD specifically, often with language that allows its use to treat epilepsy in all or specific cases, according to procon.org.

The Kentucky law, for instance, only allows prescriptions by hospitals and clinics linked to public universities. A law in Missouri, according to procon.org, only allows the use of certain kinds of “cannabis oil” for intractable epilepsy and “requires a neurologist to determine that the patient did not respond to at least three treatment options to be eligible to use the marijuana extract.”

 

In Oklahoma, an anticannabis law excludes “from the definition of marijuana ‘any federal Food and Drug Administration–approved cannabidiol drug or substance.’ ”

North Carolina attorney Rod Kight, who represents businesses in the cannabis industry, predicts that “once the federal government [via the DEA] changes the law, the states will fall in line. The only thing that would prevent them would be a lack of understanding of what’s going on.”

Meanwhile, many sellers of CBD-based products continue to promote their use as treatments for epilepsy and many other conditions.

Legal exceptions made by states for FDA-approved CBD-based drugs could affect access to unapproved CBD-based products. It’s also possible that Epidiolex could hurt sellers of cannabis products in other ways. “They’ll now have to compete with a drug made to a specific strength and purity, and that’s probably going to have some impact on their business,” William J. McNichol, adjunct professor at Rutgers Law School, Camden, N.J., said in an interview. “If you have a choice, are you going to choose aspirin I made myself in my artisan aspirin factory that the FDA never saw? Or aspirin made by Bayer?”

But there’s a big obstacle to the widespread use of Epidiolex, especially for off-label uses for other types of epilepsy: It’s expected to cost $25,000 a year.

What’s next? For one, the FDA’s approval of Epidiolex seems likely to open the door for more cannabis-based medications. “The FDA is quite open to evaluating cannabinoid-based medicines as long as they go through their process,” said Mr. Schultz, the Greenwich Biosciences spokesman.

As for the legal front, Mr. Kight said potential changes in federal law could expand the legality of cannabis-based products by allowing them when they’re derived from “industrial hemp.” This could mean that patients with epilepsy will have more legal ways to buy CBD products.

Regardless of the legal situation, Mr. Kight said, “a physician can explain to patients across the board about how CBD might benefit them. The physician can say there are extract products that are out there and available over the counter.”

As for the maker of Epidiolex, GW Pharmaceuticals is recruiting for a phase 3 trial of a cannabinoid treatment for tuberous sclerosis complex, Mr. Schultz said. The results are due in the first half of 2019.

The company is also conducting cannabinoid research in the areas of autism and Rett syndrome, Mr. Schultz said, adding: “We also have done work in schizophrenia and in oncology and glioblastoma.”

Mr. Kight disclosed that he represents companies in the cannabis industry. Dr. French is president of the Epilepsy Study Consortium and disclosed working with multiple drug makers in the epilepsy field, including GW Pharmaceuticals. Dr. French receives fixed compensation by the consortium and is not paid by any pharmaceutical company. Mr. McNichol reported no relevant disclosures.

 

The first-ever federal approval of a marijuana-derived drug, Epidiolex (cannabidiol), comes with a cloud of complications.

The drug, approved by the Food and Drug Administration in June to treat Lennox-Gastaut syndrome and Dravet syndrome in patients aged 2 years or older, is expected to be so expensive that those seeking it for off-label uses may join those who buy unregulated cannabis products on the open market. And legal complications could prevent some neurologists from prescribing Epidiolex at its planned fall launch.

Epidiolex is an oral solution of purified cannabidiol (CBD), a component of the marijuana plant that does not make people high.

According to the results of a phase 3 trial in patients with Lennox-Gastaut syndrome published earlier this year, median monthly drop seizures fell by 43.9% in a Epidiolex group compared with 21.8% in a placebo group (Lancet. 2018;391[10125]:1085-96). A 2017 study found that patients with Dravet syndrome who took the drug had fewer median convulsive seizures per month, dropping from 12.4 to 5.9, while there was barely a difference for the placebo group (N Engl J Med. 2017;376:2011-20). In both trials, patients received Epidiolex as an add-on treatment.

“There definitely are side effects such as sleepiness, abnormal liver function values, and other things that people will have to watch out for, such as interactions with other drugs,” Jacqueline A. French, MD, professor of neurology at New York University and chief scientific officer of the Epilepsy Foundation, said in an interview. “But it will definitely be a great benefit to some and a benefit to many.”

It’s not clear how the drug works, she said.

Greenwich Biosciences, the U.S. subsidiary of British drug maker GW Pharmaceuticals, plans to market Epidiolex this fall, spokesman Steve Schultz said in an interview. First, however, the U.S. Drug Enforcement Agency must reschedule the drug so it doesn’t fall under federal antimarijuana laws. It has 90 days after the FDA approval to do so, and its rescheduling is expected.

Then the focus will turn to the states, which have a crazy quilt of laws about marijuana and its medical use. “We’ve worked with the state legislators to enact laws or to modify laws to allow for our medicine to be made available,” Mr. Schultz said. “There may be one or two that we’re still working on.”

At issue: States and the District of Columbia have a wide variety of laws that appear to affect the sale of Epidiolex.

According to the website procon.org, 30 states and the District of Columbia allow medical marijuana, although the laws vary regarding what is allowed and do not address cannabis-derived pharmaceuticals.

Another 17 states have laws that address CBD specifically, often with language that allows its use to treat epilepsy in all or specific cases, according to procon.org.

The Kentucky law, for instance, only allows prescriptions by hospitals and clinics linked to public universities. A law in Missouri, according to procon.org, only allows the use of certain kinds of “cannabis oil” for intractable epilepsy and “requires a neurologist to determine that the patient did not respond to at least three treatment options to be eligible to use the marijuana extract.”

 

In Oklahoma, an anticannabis law excludes “from the definition of marijuana ‘any federal Food and Drug Administration–approved cannabidiol drug or substance.’ ”

North Carolina attorney Rod Kight, who represents businesses in the cannabis industry, predicts that “once the federal government [via the DEA] changes the law, the states will fall in line. The only thing that would prevent them would be a lack of understanding of what’s going on.”

Meanwhile, many sellers of CBD-based products continue to promote their use as treatments for epilepsy and many other conditions.

Legal exceptions made by states for FDA-approved CBD-based drugs could affect access to unapproved CBD-based products. It’s also possible that Epidiolex could hurt sellers of cannabis products in other ways. “They’ll now have to compete with a drug made to a specific strength and purity, and that’s probably going to have some impact on their business,” William J. McNichol, adjunct professor at Rutgers Law School, Camden, N.J., said in an interview. “If you have a choice, are you going to choose aspirin I made myself in my artisan aspirin factory that the FDA never saw? Or aspirin made by Bayer?”

But there’s a big obstacle to the widespread use of Epidiolex, especially for off-label uses for other types of epilepsy: It’s expected to cost $25,000 a year.

What’s next? For one, the FDA’s approval of Epidiolex seems likely to open the door for more cannabis-based medications. “The FDA is quite open to evaluating cannabinoid-based medicines as long as they go through their process,” said Mr. Schultz, the Greenwich Biosciences spokesman.

As for the legal front, Mr. Kight said potential changes in federal law could expand the legality of cannabis-based products by allowing them when they’re derived from “industrial hemp.” This could mean that patients with epilepsy will have more legal ways to buy CBD products.

Regardless of the legal situation, Mr. Kight said, “a physician can explain to patients across the board about how CBD might benefit them. The physician can say there are extract products that are out there and available over the counter.”

As for the maker of Epidiolex, GW Pharmaceuticals is recruiting for a phase 3 trial of a cannabinoid treatment for tuberous sclerosis complex, Mr. Schultz said. The results are due in the first half of 2019.

The company is also conducting cannabinoid research in the areas of autism and Rett syndrome, Mr. Schultz said, adding: “We also have done work in schizophrenia and in oncology and glioblastoma.”

Mr. Kight disclosed that he represents companies in the cannabis industry. Dr. French is president of the Epilepsy Study Consortium and disclosed working with multiple drug makers in the epilepsy field, including GW Pharmaceuticals. Dr. French receives fixed compensation by the consortium and is not paid by any pharmaceutical company. Mr. McNichol reported no relevant disclosures.