According to a story from GlobeNewswire, the biopharmaceutical company GW Pharmaceuticals plc recently announced that its CBD oral solution Epidiolex® has recently been approved as a treatment for seizures associated with the rare disease tuberous sclerosis complex. The medication is cleared for use in patients age one or older. Epidiolex was previously approved for seizures associated with Lennox-Gastaut syndrome and Dravet syndrome, but the age range has now been expanded to cover patients age one or older with those disorders as well.
About Tuberous Sclerosis Complex
Tuberous sclerosis complex (TSC) is a rare genetic disease that causes a variety of symptoms. It is most distinguished by the appearance of non-cancerous tumors, which grow in the brain, lungs, eyes, liver, skin, and other organs. The disease is triggered by mutations affecting the TSC1 or TSC2 genes, which code for proteins that normally suppress the appearance of tumors. Tuberous sclerosis complex can inflict a variety of symptoms, such as intellectual disability, skin changes, disease affecting the lungs or kidneys, behavioral abnormalities, developmental delays, and seizures. The broad systemic impacts of the disease require frequent management and monitoring of tumors; a variety of specialist expertise may be necessary. Outcomes vary, but with good management most patients have a normal life expectancy. Many patients with mild symptoms remain undiagnosed. To learn more about tuberous sclerosis complex, click here.
A New Treatment Option
The drug has been in use since 2018 as a treatment for Dravet syndrome and Lennox-Gastaut syndrome, which, like tuberous sclerosis complex, are genetic causes of epilepsy that can be severe and often do not respond to other therapies. The approval of Epidiolex in this indication follows promising results from a phase 3 clinical trial in which the oral solution was able to reduce seizures in 48 percent of patients compared to 24 percent in the placebo group.
The trial tested a dose of 25 mg/kg a day and this is the recommended dose as approved by the US Food and Drug Administration (FDA). For some tuberous sclerosis complex patients, seizures can be their most frequent and challenging symptom. These seizures can also be very dangerous with patients being at a higher risk of status epilepticus, defined as a seizure lasting five minutes or more or two seizures within five minutes. These long seizures increase the patient’s risk of death.
The availability of Epidiolex will give these patients a new resource for combating seizures.