In a Press Release published July 31, 2020, the Food and Drug Administration (FDA) approves Epidiolex (cannabidiol) Oral Solution to Treat Seizures Associated with Tuberous Sclerosis Complex (TSC). It was confirmed that Epidiolex can now be prescribed for seizures associated with TSC in patients one year or older.
The FDA previously approved Epidiolex as the first drug that contains a purified drug substance derived from cannabis. In 2018, after reviewing three clinical trials, the CBD solution was found to be a safe and effective treatment for patients aged two years or older who were suffering from Lennox-Gastaut and Dravet syndrome, being severe forms of epilepsy.
FDA has now approved the third indication – TSC related seizures, to the list conditions treatable by Epidiolex and expanded the patient age range to allow children over the age of one suffering with Lennox-Gastaut or Dravet syndrome to access the treatment.
“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 in a statement. “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.”
GW Pharmaceuticals, the manufacturer of Epidiolex, announced on Monday, August 3, 2020, confirming that the FDA had approved EPIDIOLEX oral solution as a treatment for seizures associated with TSC.
“FDA approval of this new indication is exciting news for those with refractory seizures due to tuberous sclerosis complex,” said Justin Gover, GW’s Chief Executive Officer in a statement. “Since EPIDIOLEX is already available to patients by physician’s prescription, patients with TSC can immediately access the medication. This label expansion, including the expansion of the age range in all approved indications, further demonstrates that the FDA process can continue to enable broader patient access to appropriately tested regulatory approved cannabinoid medicines. It also provides hope for these patients and their families and is yet another important milestone for EPIDIOLEX as a first-in-class antiepileptic drug.”
About Tuberous Sclerosis Complex (TSC)
According to Tuberous Sclerosis Alliance, TSC is Affecting approximately 50,000 in the United States and 1 million worldwide. TSC is a genetic disorder that causes non-cancerous tumors to form in vital organs; it is also the leading genetic cause of epilepsy and autism.
“Up to 85 percent of those affected by TSC experience seizures at some point in their lifetime,” explained TS Alliance President & CEO Kari Luther Rosbeck in a statement. “Unfortunately, existing medications don’t always effectively control them. Our organization and the TSC community certainly welcome a new option such as Epidiolex, which is the second FDA-approved drug specific to TSC.”
At least two children born each day will develop TSC, with an estimated rate of one in 6,000 newborns. People with TSC may experience a variety of seizure types. One of the most common is infantile spasms that typically present in the first year of life. The condition causes mostly benign tumors to grow in vital organs of the body (such as the brain, heart, skin, eyes, lungs, and kidneys) and is a leading cause of genetic epilepsy. People with TSC may experience a variety of seizure types. TSC is associated with a greater risk of autism and intellectual disabilities, with its severity varying widely. In some patients, the symptoms can be very mild, while others may experience serious complications. Many people with TSC live healthy and independent lives while enjoying challenging professions such as doctors and lawyers. The severity of the various aspects of TSC varies widely between patients, even between identical twins.
EPIDIOLEX (cannabidiol) oral solution, a pharmaceutical formulation of highly purified cannabidiol (CBD), is an anti-epileptic medication with a novel mechanism of action. It is the first prescription, plant-derived cannabis-based medicine approved by the FDA. EPIDIOLEX has been indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome, or tuberous sclerosis complex (TSC) in patients one year of age and older.
Further information about EPIDIOLEX is available at Epidiolex.com.
The National Institute of Standards and Technology (NIST) has announced that a new program launched to standardize Cannabis testing methods and to aid commercial and forensic laboratories to accurately measure chemical compounds in marijuana, hemp, and other cannabis products.
The aim of the Cannabis Quality Assurance Program (CannaQAP) is to achieve accuracy in product labeling and help laboratories to distinguish between hemp and marijuana.
While the labels on most cannabis products illustrate the concentration of tetrahydrocannabinol (THC) and cannabidiol (CBD), without official testing methodology, laboratories can often differ in their testing techniques, in turn, providing ’unreliable’ results.
It is therefore primarily the inconsistencies in testing results that the researchers at NIST are looking to address in the course of the program.
“When you walk into a store or dispensary and see a label that says 10% CBD, you want to know that you can trust that number.” —NIST research chemist Brent Wilson
To achieve the program’s objective, NIST is looking to partner up with a range of hemp testing laboratories. The first point of the CannaQAP effort will consist of NIST sending out hemp oil samples to the participating laboratories and asking them to measure the total of CBD and THC using their routine testing methods. Each lab will then test the oils and relay its results and techniques back to the NIST researchers. It is expected that further exercises will include the testing of plant material samples.
The data collected from the participating laboratories will then be evaluated. NIST is looking to publish the data anonymously so that the participating laboratories’ names are not revealed. Research chemist Melissa Phillips has stated: “Anonymity means that labs don’t have to worry about how their performance will be viewed. Our goal is to help labs improve, not to call them out.”
While the laboratories will not be named, the results will show how much variability there is between them. Furthermore, NIST is looking to publish the correct measurements, so that each laboratory can see how accurate its measurements were and how it performed compared to its peers.
Once NIST researchers can review the results, and evaluate the scope of the inconsistencies, they will be in a much better position to issue guidance on best practices for cannabis testing.
NIST has said it will be developing a standard hemp reference material, which will provide accurate measurement values that labs can use to validate their testing methods. One reason these measurements vary so much is that there are currently no reference materials for cannabis.
Finally, it is understood that the new federal CannaQAP endeavour might go beyond legal hemp products. “NIST is also planning to conduct future exercises with ground hemp and possibly marijuana,” the agency has indicated. “Those exercises will involve measuring a larger number of compounds, including terpenes—the chemicals that give different strains of marijuana their distinct aromas—and compounds that people don’t want in their cannabis such as fungal toxins, pesticides, and heavy metals. Future exercises may also include extracts, concentrates, distillates, and edibles.”
We will continue following the program and will provide updates as soon as new data comes to light. Be sure to subscribe to our newsletter to stay in the know. Laboratories that are interested in participating in the program can find more information and register online.
FDA Issues Draft Guidance on CBD Clinical Research and Submits CBD Enforcement Policy to OMB for Review
July 2020 has been a busy month in the CBD world whereby FDA issues draft guidance on CBD clinical research and submits CBD enforcement policy to OMB for review.
After much anticipation, the FDA has begun to address its policies on CBD. On July 21, 2020, FDA issued draft guidance on clinical research involving CBD-containing investigational drugs and other cannabis-derived compounds. Further, on July 22, 2020, the long-awaited draft guidance on CBD enforcement policy was sent to the Office of Management and Budget (OMB) for a review.
To date, FDA’s movement has not officially changed the existing position on the legal and regulatory status of various CBD-containing consumer products, but more is expected. Still, the recent reports to Congress and draft guidance on investigations involving CBD and other cannabis-derived compounds do provide us with a glimpse of FDA’s current reasoning surrounding CBD.
Clinical Research Draft Guidance
On July 21, 2020, FDA issued draft guidance on quality considerations for clinical research where cannabis-derived compounds are concerned. The guidance titled “Cannabis and Cannabis-Derived Compounds: Quality Considerations for Clinical Research” builds off previous guidance FDA had issued regarding the quality and regulatory considerations that govern the development and FDA approval of cannabis products. The draft guidance highlights the following key points :
1. Sourcing of Cannabis
For many years, the only domestic source of cannabis for legal clinical research was the National Institute on Drug Abuse (NIDA) Drug Supply Program (DSP), but with the changes made by the 2018 Farm Bill, hemp can serve as a source for cannabis cannabis-derived compounds for drug development. This change gives the investigational drug sponsors of clinical studies new options that do not involve the NIDA DSP.
2. Quality Considerations
Sponsors of cannabis-derived compounds will be expected to provide sufficient information to ensure the identity, quality, purity, and potency or strength of the investigational product. Furthermore, there will be a requirement to provide quantitative data regarding phytochemicals present in their proposed product, such as cannabinoids, terpenes, and flavonoids. The guidance goes on to list several helpful resources from which sponsors may obtain more information about specific methodologies and other related insights. The guidance clarifies that cannabis is held to the same regulatory standards as any other botanical raw material, botanical drug substance, or botanical drug product; therefore, any FDA guidance that pertains to such items will also be helpful.
3. THC Calculation
The guidance acknowledges some of the challenges of calculating THC and notes the importance of consulting the DEA before commencing drug-development activities. Amongst other things, the FDA recommends that sponsors calculate the THC level in their product candidates early in the development process to gain insight into its potential control status.
Cannabidiol Enforcement Policy Draft Guidance
On July 22, 2020, the FDA sent its draft Cannabidiol Enforcement Policy to OMB for review. The scope and the contents of the document titled “Cannabidiol Enforcement Policy; Draft guidance for Industry” are not yet publicly available, but the industry is eagerly anticipating the long-awaited guidance on CBD enforcement.
While the full extent of the FDA’s intended enforcement consideration will be clear only when guidance is finally released, we expect, like many others, that FDA will focus its efforts on consumer products that carry the most significant risk to public health. Therefore, we anticipate that the enforcement guidance will focus on lawful marketing and transparency.
We will continue to monitor the FDA’s issuance of the enforcement guidance and update as soon as it passes the OMB review. Make sure to sign up to our newsletter for further updates.