Epidiolex (cannabidiol) for Lennox-Gastaut syndrome
Fact-checked by What is Epidiolex for Lennox-Gastaut syndrome?
Epidiolex (cannabidiol) is an oral therapy approved to treat seizures associated with Lennox-Gastaut syndrome (LGS) and certain other conditions.
LGS is a rare form of childhood-onset epilepsy that’s characterized by recurrent, medication-resistant seizures.
The active ingredient in Epidiolex is a highly purified form of cannabidiol, the major nonintoxicating component of the cannabis plant. While its therapeutic mechanism is not fully understood, the treatment is thought to help control excessive and abnormal brain activity that causes seizures. It may do so in a few different ways, including by modulating calcium levels in brain cells.
The medication, which comes in the form of an oral solution, is sold by Jazz Pharmaceuticals. In the European Union, it is marketed under the brand name Epidyolex. The medication is also approved to treat seizures in people with Dravet syndrome and tuberous sclerosis complex.
Therapy snapshot
| Brand name | Epidiolex |
| Chemical name | Cannabidiol |
| Usage | Used to treat seizures in people with LGS |
| Administration | Oral solution |
Who with LGS can take Epidiolex?
In the U.S., Epidiolex is approved for treating seizures in people with LGS ages 1 and older. The medication is also approved for LGS in the EU and Canada, although specific indications may vary.
It is contraindicated, or should not be used, by people with a history of severe immune reactions (hypersensitivity) to cannabidiol or any other ingredients in the medication.
How is Epidiolex administered in LGS?
Epidiolex comes as a strawberry-flavored oral solution to be taken twice daily. If necessary, the medication can be administered through a feeding tube.
The recommended starting dose in LGS is 2.5 mg/kg twice daily (5 mg/kg daily). Based on individual responses and tolerability, the dose can be slowly increased to a maximum of 10 mg/kg twice daily (20 mg/kg daily).
Dose adjustments may be required for people with moderate or severe liver impairments. Liver health must be evaluated prior to starting treatment.
Epidiolex in LGS syndrome clinical trials
Epidiolex’s approval for LGS was supported by two placebo-controlled Phase 3 clinical trials: GWPCARE4 (NCT02224690) and GWPCARE3 (NCT02224560). Both involved patients ages 2-55 whose seizures were not adequately controlled on at least one antiseizure medication.
- In both studies, Epidiolex (10 or 20 mg/kg daily) was associated with significant reductions in the frequency of drop seizures — seizures that cause a person to slump over or fall down — over a 14-week treatment period compared with a four-week run-in period before treatment was started.
- The median percent reduction in monthly seizure frequency in either study was approximately 40% with Epidiolex, compared with about 15%-20% in the placebo group.
- Greater improvements on a caregiver-rated measure of change were also observed with Epidiolex.
Epidiolex side effects
The most common side effects of Epidiolex in people with LGS or Dravet syndrome include:
- drowsiness (somnolence)
- decreased appetite
- diarrhea
- liver enzyme elevations
- fatigue and a lack of energy
- a general feeling of being unwell
- rash
- infections
- difficulty sleeping (insomnia), poor sleep quality, or other sleep problems
The drug also comes with warnings for potentially serious adverse events, including:
- liver injury, particularly in people also using the antiseizure medications valproate or clobazam
- somnolence or sedation
- suicidal thoughts or behaviors
- hypersensitivity reactions
If these events occur, Epidiolex may need to be discontinued or the dose adjusted. Individuals should not drive or operate machinery until they know how their body reacts to the medication.
Epidiolex may interact with other medications, including antiseizure drugs, which may increase the risk of certain side effects. Some of these medications may need to be discontinued or the dose adjusted if being used with Epidiolex.
If discontinued, the medication’s dose should be gradually reduced. Abruptly stopping the treatment could raise the risk of increased seizure frequency and a medical emergency known as status epilepticus.
Lennox-Gastaut Syndrome News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
