FDA approves new cannabis extract for treatment of severe epilepsy

Today, the US FDA announced the approval of the pharmaceutical company GW's Epidiolex (cannabidiol) oral liquid for the treatment of two rare and serious epilepsy - Dravet syndrome and Lennox-Gastaut syndrome, which is the first FDA-approved purification New drug for cannabis extract.

It is understood that Dravet syndrome and Lennox-Gastaut syndrome are two rare types of epilepsy diseases. Dravet syndrome (DS), also known as severe myoclonic epilepsy in infants, is a rare cause mainly caused by genetic factors. Progressive epilepsy encephalopathy has the characteristics of early onset age, multiple types of seizures, high frequency of seizures, serious mental damage, and poor drug treatment. The disease has a poor prognosis and almost all children have cognitive impairment. Lennox-Gastaut Syndrome (LGS) is a type of age-related cryptogenic or symptomatic systemic epilepsy syndrome, age-dependent epilepsy. It is characterized by early onset of onset, onset in early childhood, various forms of seizures, mental development affected, and difficult treatment, which is a serious type of epilepsy.

Epidiolex is an oral, high-purity Cannabidiol (CBD) extract liquid preparation. CBD is a non-psychotic ingredient from cannabis plants that has a variety of pharmacological effects on the nervous system. Numerous studies have shown that CBD has significant anti-epileptic and anticonvulsant activity, with fewer side effects than existing anti-epileptic drugs.

The potential of Epidiolex for the treatment of epilepsy was validated in three randomized, double-blind, placebo-controlled clinical trials. These clinical programs enrolled 516 patients and showed that Epidiolex was used in combination with other drugs to effectively reduce the frequency of seizures compared with placebo. Previously, for its therapeutic potential, the new drug was awarded the FDA-issued fast-track qualification (for Dravet syndrome) and orphan drug qualification (for both epilepsy). After the listing application was accepted, the US FDA also granted its priority for review. In addition, the FDA also granted Epidiolex in 2016 to treat orphan drug status in rare hereditary tuberous sclerosis (TSC). TSC can cause non-malignant tumors in many different organs, of which the brain and skin are the most common affected tissues. The most common symptom of TSC is epilepsy, which occurs in 80%-90% of patients, and is also a disease in TSC patients. An important cause of death.

Billy Dunn, head of neurology products at the US FDA's Center for Drug Evaluation and Research, for the approval of Epidiolex: it brings the first to Dravet syndrome patients in addition to another important treatment for patients with Lennox-Gastaut syndrome. The approval of new drugs will bring important and much-needed improvements to their situation.