A Family’s Perspective – “The Brutality of Sepsis will Haunt Us for the Rest of Our Lives”
CBD Drug For Tuberous Sclerosis: Caregivers Continue Epidiolex Treatment Citing Seizure Reduction And Cognitive Improvements
The majority of caregivers of patients with tuberous sclerosis complex (TSC) say they plan to continue Epidiolex treatment, citing benefits like reduced seizure frequency and improvements in cognition and communication. That's according to data from the BECOME-TSC (BEhavior, COgnition, and More with Epidiolex) caregiver survey, announced by Jazz Pharmaceuticals plc JAZZ.
New data on Epidiolex is being presented at the American Epilepsy Society (AES) 2024 Annual Meeting, being held December 6-10 in Los Angeles, California via nine company-sponsored posters.
Epidiolex is the only FDA-approved prescription CBD treatment for severe forms of epilepsy. It was developed and produced by GW Pharmaceuticals, which was acquired by Jazz Pharmaceuticals in 2021.
Epidiolex was first approved for treating seizures connected to Lennox-Gastaut syndrome and Dravet syndrome. In 2022, Epidiolex was approved for treating seizures related to tuberous sclerosis complex, a rare genetic disorder that causes non-cancerous tumors to grow in many parts of the body, including the brain, skin, heart, eyes, kidneys and lungs.
Read Also: The Only FDA-Approved CBD Treatment For Severe Epilepsy Completes Key Phase 3 Trial In Japan Despite Missed Primary Goal
"Our real-world data presentations at AES 2024, including novel findings from the BECOME-LTC, BECOME-TSC and EpiCom studies, demonstrate the meaningful impact of Epidiolex in the treatment of patients with rare epilepsies," said Sarah Akerman, MD, head of neuroscience global medical and scientific affairs of Jazz Pharmaceuticals. "These findings increase our understanding of Epidiolex's benefits beyond seizure control, addressing unmet needs across a range of epilepsy syndromes for people living with rare epilepsies and demonstrating reproducibility and consistency of effect across different populations."
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JAZZJazz Pharmaceuticals PLC
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Epidiolex Delivers Positive Results For A Third Rare Condition Called TSC
Epidiolex reports positive results for its drug when treating tuberous sclerosis complex (TSC) a rare genetic condition. The condition causes mostly benign tumors to grow in vital organs of the body including the brain, skin, heart, eyes, kidneys and lungs and is a leading cause of genetic epilepsy.
Epidiolex was the leading medical marijuana drug originally produced by GW Pharmaceuticals to treat the rare pediatric epilepsy diseases – Dravat Syndrome and Lennox Gastaut. GW Pharmaceuticals was later acquired by Jazz Pharmaceuticals (NASDAQ: JAZZ). Epidiolex was the first medical non-synthetic marijuana drug to go through the highly regulated FDA study trials. It has been instrumental in reducing the stigma of medical marijuana by demonstrating significant reductions in seizures for children with these diseases.
Real-world data"Our real-world data presentations at AES 2024, including novel findings from the BECOME-LTC, BECOME-TSC and EpiCom studies, demonstrate the meaningful impact of Epidiolex in the treatment of patients with rare epilepsies," said Sarah Akerman, MD, head of neuroscience global medical and scientific affairs of Jazz Pharmaceuticals. "These findings increase our understanding of Epidiolex's benefits beyond seizure control, addressing unmet needs across a range of epilepsy syndromes for people living with rare epilepsies and demonstrating reproducibility and consistency of effect across different populations." BECOME-TSC refers to BEhavior, COgnition, and More with the Epidiolex caregiver survey.
The company will present the results at the American Epilepsy Society (AES) 2024 Annual Meeting, which will be held this week in Los Angeles, California. The company reported some of the findings as follows:
A three-month study demonstrated improvements after an Epidiolex treatment in the severity of behavioral problems in patients with TSC as reported by the TAND Self-Report Quantified Checklist and Aberrant Behavior Checklist.
Two updated analyses showed that, of 55 caregivers who completed the survey, 89% planned to continue Epidiolex treatment for their loved one. The most important reasons for continuing Epidiolex included seizure and non-seizure benefits such as reduced seizure frequency and severity/duration and TAND-related improvements in cognition and language/communication.
Results from the BECOME-LTC (BEhavior, COgnition, and More with Epidiolex in the Long-Term Care Setting) survey found that, among 102 nurses surveyed, 85% reported a reduction in the overall frequency of any seizure type after Epidiolexinitiation, with 49% reporting a greater than 50% reduction.
Results from CARE-EpiC (Caregiver Analysis of Real-world Epidiolex in Epilepsy Context), a cross-sectional caregiver survey, demonstrated reduced caregivers' need for additional support of their dependents' physical, emotional, and behavioral care after Epidiolex initiation and characterized improvements in their dependents' well-being as well as caregivers' experiences.
In the TSC group, Epidiolex was associated with a median reduction from baseline of 51%–87% in focal seizures and 44%–87% in total seizures. In the non-TSC group, Epidiolex was also associated with a median reduction from baseline of 46%–75% in focal and 46%–74% in total seizures.
Green Market Report wrote in November that Epidiolex, accounted for $251.5 million in revenue for the company's third quarter, up from $213.7 million the same quarter a year ago. For the past nine months ending in September, Epidiolex has generated $697 million in sales – an increase of 15% over the previous year. Epidiolex, which is now available in 35 countries, was second in sales only to Xywav, Jazz's sleep aid drug, which sold $388.4 million in the quarter.
The company has been facing patent lawsuits from other medical marijuana companies including Teva Pharmaceuticals, Inc. And Ascent Pharmaceuticals. Some of the original claims to create a generic version of the drug dating back to 2022 were dismissed but others were allowed to move forward. Jazz managed to reach settlements with some parties, but then new cases were filed. A trial is scheduled for September 2025.
Separately, On November 20, 2024, Jazz Pharmaceuticals announced that the FDA accelerated approval of its Ziihera (zanidatamab-hrii) 50mg/mL for injection for IV for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC). The FDA granted accelerated approval for this indication based on overall response rate and duration of response.
All About Epidiolex
Epidiolex (cannabidiol) is a prescription drug used to manage seizures caused by certain disorders. Epidiolex comes as a liquid that you swallow.
Specifically, Epidiolex is used to help manage seizures caused by:
For more information about the specific uses of Epidiolex, see the "What is Epidiolex used for?" section.
Epidiolex basicsThe active ingredient in Epidiolex is cannabidiol (CBD). An active ingredient is what makes a drug work.
It's available as a liquid solution that you swallow.
Epidiolex is only available in brand-name form. Currently, there are no generic forms of Epidiolex available.
Read on to learn more about Epidiolex and its uses, side effects, and other information.
Epidiolex is used to help manage seizures caused by the following conditions:
For managing seizures from these conditions, Epidiolex can be used in adults and children ages 12 months and older.
Seizures may occur from unusual signaling in the electrical activity of the brain. They can cause confusion, uncontrollable movements, and loss of consciousness.
Epidiolex's mechanism of action for managing seizures is unknown. "Mechanism of action" refers to how a drug works to treat a condition.
Like most drugs, Epidiolex may cause mild or serious side effects. The following lists describe some of the more common side effects Epidiolex may cause. These lists don't include all possible side effects.
Keep in mind that side effects of a drug can depend on:
Your doctor or pharmacist can tell you more about the potential side effects of Epidiolex. They can also suggest ways to help reduce side effects.
Mild side effectsHere's a list of some of the mild side effects that Epidiolex can cause. To learn about its other mild side effects, talk with your doctor or pharmacist, or read Epidiolex's prescribing information.
Mild side effects of Epidiolex that have been reported include:
Mild side effects of drugs may go away within a few days to a couple of weeks. But if they become bothersome, talk with your doctor or pharmacist.
Serious side effectsSerious side effects from Epidiolex can occur, but they aren't common. If you have serious side effects from Epidiolex, call your doctor right away. But if you think you're having a medical emergency, you should call 911 or your local emergency number.
Serious side effects of Epidiolex that have been reported include:
Your doctor will recommend the dosage of Epidiolex that's right for you. Here are commonly used dosages, but always take the dosage your doctor prescribes.
Form and concentrationEpidiolex is available as a liquid solution that you swallow.
Epidiolex comes in one concentration: 100 milligrams per milliliter of solution (mg/mL).
Recommended dosageYou'll take a dose of Epidiolex twice per day. Your doctor will calculate your dose based on your body weight in kilograms (kg). One kilogram is equal to about 2.2 pounds (lb).
Epidiolex's manufacturer offers a dosing calculator, which you can use to check the dosage your doctor prescribed.
Dosage for Dravet syndrome or Lennox-Gastaut syndromeDoctors may prescribe Epidiolex to help manage seizures caused by Dravet syndrome or Lennox-Gastaut syndrome. The recommended starting dose for this condition is 2.5 mg per kg of body weight (mg/kg). You'll take this twice per day, for a total daily dose of 5 mg/kg.
The typical recommended maintenance dosage of Epidiolex for these conditions is 5 mg/kg twice daily (10 mg/kg total).
Depending on how your condition responds to Epidiolex, your doctor can increase your dosage to a maximum of 10 mg/kg twice per day (20 mg/kg total).
Dosage for tuberous sclerosis complexEpidiolex can be used to help treat seizures caused by tuberous sclerosis complex. When prescribed this way, the recommended starting dose is 2.5 mg/kg twice per day (5 mg/kg total).
Each week, your doctor will likely increase your dose by 2.5 mg/kg twice daily (5 mg total). They'll increase your dose until you reach the recommended maintenance dose of 12.5 mg/kg twice per day, for a total daily dose of 25 mg/kg.
How to takeEpidiolex is available as a liquid solution that you swallow. You should always use the measuring syringes that come with Epidiolex. This is the best way to make sure that your dose is accurate.
It's recommended that you discard any Epidiolex that you don't use within 12 weeks after opening the bottle.
You should not suddenly stop taking Epidiolex. It's possible that suddenly stopping treatment can increase your risk of seizures. If you need to stop taking Epidiolex, talk with your doctor. They'll lower your dosage gradually to help decrease your risk of seizures.
Taking Epidiolex with other drugsIn many cases, people who have a seizure disorder take multiple medications to treat their condition. So your doctor may recommend taking Epidiolex along with other seizure medications. Examples include levetiracetam (Keppra) and topiramate (Topamax).
Before you start taking Epidiolex, your doctor will determine the best treatment options for you.
Questions about taking EpidiolexHere are some common questions about Epidiolex's dosing.
Do not take more Epidiolex than your doctor prescribes. Using more than this can lead to serious side effects.
What to do in case you take too much EpidiolexCall your doctor if you think you've taken too much Epidiolex. You can also call 800-222-1222 to reach America's Poison Centers or use its online resource. But if you have severe symptoms, immediately call 911 or your local emergency number. Or go to the nearest emergency room.
Certain factors can affect whether Epidiolex is a good treatment option. These include:
These factors and others are discussed here in more detail.
InteractionsTaking a medication with certain vaccines, foods, and other things can affect how the medication works. These effects are called interactions.
Before taking Epidiolex, be sure to tell your doctor about all medications you take, including prescription and over-the-counter types. Also, describe any vitamins, herbs, or supplements you use. Your doctor or pharmacist can tell you about any interactions these items may cause with Epidiolex.
For information about drug-condition interactions, see the "Warnings" section.
Interactions with drugs or supplementsEpidiolex can interact with certain kinds of drugs or specific drugs. These include:
This list does not contain all the drugs that may interact with Epidiolex. Your doctor or pharmacist can tell you more about these interactions and any others that may occur when using Epidiolex.
Other interactionsIn addition, Epidiolex can interact with certain supplements, such as St. John's wort and caffeine.
This medication can also interact with alcohol. For more information, see the "Epidiolex and alcohol" section.
Before you start taking Epidiolex, tell your doctor about any supplements that you take. They will let you know whether your supplements are safe to take with this medication.
WarningsEpidiolex may not be right for you if you have certain medical conditions. These are known as drug-condition interactions. Other factors may also affect whether Epidiolex is a good treatment option for you.
Talk with your doctor about your health history before you take Epidiolex. Factors to consider include those in the following list.
Depression, other mood disorders, or suicidal thoughts or behaviors: Epidiolex can increase the risk of suicidal thoughts or behaviors. People who have depression, mood problems, or suicidal thoughts or behaviors have an even higher risk of this side effect.
If you have a personal or family history of any of these mental health conditions, tell your doctor before you take Epidiolex. This will help them determine whether Epidiolex is safe for you to take. In some cases, they may recommend a different treatment option for you.
Liver problems: Epidiolex may cause liver damage. If you already have liver problems, taking Epidiolex can worsen your condition. Tell your doctor about any liver problems you have before starting Epidiolex treatment. They can check your liver before and during treatment. Depending on the condition of your liver, your doctor may recommend a different treatment option for you.
Allergic reaction: If you've had an allergic reaction to Epidiolex or any of its ingredients, including sesame seed oil, your doctor will likely not prescribe Epidiolex. Ask them what other medications are better options for you.
Epidiolex and alcoholYour doctor may recommend avoiding alcohol while you're taking Epidiolex. This medication may cause you to feel sleepy or less alert, and so can alcohol. As a result, drinking alcohol during treatment may increase your risk of such side effects.
If you drink alcohol, ask your doctor about how much may be safe for you to consume while taking Epidiolex.
Pregnancy and breastfeedingIt's not known whether Epidiolex may be safe to take during pregnancy or while breastfeeding.
At this time, there haven't been studies of the drug during pregnancy. It's also not known whether Epidiolex may pass into breast milk or its effects on a breastfeeding child.
If you're pregnant or breastfeeding or planning to become pregnant or breastfeed, talk with your doctor before taking Epidiolex. They'll discuss with you the potential risks and benefits of taking this drug during pregnancy or while breastfeeding.
A pregnancy registry collects information about the effects of a drug when taken during pregnancy. This can help determine the drug's effects on a developing fetus or someone who is pregnant. If you take Epidiolex while pregnant, consider enrolling in the pregnancy registry for antiseizure drugs. For information, call 888-233-2334 or visit the registry website.
Find answers to some commonly asked questions about Epidiolex.
Is Epidiolex a controlled substance?No, Epidiolex currently isn't a controlled substance. Controlled substances are drugs regulated by the United States government because they may cause dependence or misuse. With dependence, a person's body becomes used to a drug and needs it to function as usual. Misuse is using a medication in a different way than prescribed, such as taking more than recommended.
Epidiolex contains CBD as its active ingredient, and some people may try to misuse it. This is because they may think the drug causes a "high," which isn't the case. So it's important to store the drug in a secure place.
In addition, be sure to keep the prescription label on your bottle of Epidiolex, especially when traveling. In the U.S., it's legal to have prescription Epidiolex. But in some states, using CBD without a prescription is illegal.
Can Epidiolex treat anxiety or schizophrenia?At this time, Epidiolex isn't approved by the Food and Drug Administration (FDA) to treat anxiety or schizophrenia. This drug is currently only approved to manage seizures caused by Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex.
One study suggests that CBD, the active ingredient in Epidiolex, may ease anxiety symptoms. But there isn't enough information yet to determine whether the drug may be safe or effective in people with anxiety.
In a study of CBD for treating schizophrenia, people taking the drug had a decrease in symptoms. But another study found that CBD didn't affect schizophrenia symptoms. More studies or clinical trials are needed to determine whether Epidiolex may be a safe or effective treatment for people with schizophrenia.
If you're interested in using Epidiolex to treat anxiety or schizophrenia, talk with your doctor. They can work with you to devise the best treatment plan for your condition. For more information about Epidiolex's approved uses, see the "What is Epidiolex used for?" section.
Is Epidiolex used for pain, migraine episodes, or autism spectrum disorder?Currently, Epidiolex is only approved by the FDA to manage seizures caused by Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex. It's not currently approved to treat pain, migraine episodes, or autism spectrum disorder (ASD).
Studies of CBD, the active ingredient in Epidiolex, suggest the drug may work for treating symptoms of migraine headaches and ASD. Results from studies of CBD as a treatment for pain have been mixed. At this time, more studies are needed to determine whether Epidiolex may be a safe and effective treatment for these conditions.
If you're interested in using Epidiolex to treat any of these conditions, talk with your doctor. They can work with you to develop the best treatment plan for your condition.
Is Epidiolex the same as CBD?Yes and no. Epidiolex contains CBD as its active ingredient. But Epidiolex is FDA approved to manage seizures in some people. Over-the-counter CBD products aren't regulated by the FDA, so they aren't approved to treat specific medical conditions.
It's also important to note that nonprescription CBD products may contain other active ingredients, including small amounts of tetrahydrocannabinol (THC). Epidiolex contains CBD as its only active ingredient.
Epidiolex and nonprescription CBD products may also differ in other ways, including cost and dosage. For more detailed information about which treatment option is right for you, talk with your doctor or pharmacist.
People may take Epidiolex or Charlotte's Web supplements to help manage certain seizure disorders. However, only Epidiolex is approved for this use.
Charlotte's Web supplements and products contain hemp, so they aren't regulated by the FDA. This means the manufacturer of Charlotte's Web can't make claims about medical uses for this product.
Another difference between Epidiolex and Charlotte's Web is the dosage form. Epidiolex is only available as a liquid solution that you swallow. Charlotte's Web comes as creams, capsules, and oils.
To learn more about how Epidiolex compares with Charlotte's Web, see the "Epidiolex vs. Charlotte's Web" section of this Epidiolex overview.
The price of prescription drugs can vary depending on many factors. These factors include what your insurance plan covers (if you have insurance) and which pharmacy you use.
If you have questions about how to pay for your prescription, talk with your doctor or pharmacist. You may also be eligible for an Epidiolex copay savings program from the drug's manufacturer.
Learn more about saving money on prescriptions.
Disclaimer: Healthline has made every effort to make certain that all information is factually correct, comprehensive, and up to date. However, this article should not be used as a substitute for the knowledge and expertise of a licensed healthcare professional. You should always consult your doctor or another healthcare professional before taking any medication. The drug information contained herein is subject to change and is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. The absence of warnings or other information for a given drug does not indicate that the drug or drug combination is safe, effective, or appropriate for all patients or all specific uses.
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