Magic cannabinoid in the treatment of pediatric intractable epilepsy! Epidiolex's fourth phase III study was successful and has been listed in the United States

November 29, 2018 / BIOON / - GW Pharma, a British pharmaceutical company, is a global leader in the research and development of plant-based cannabinoid treatment products, committed to the discovery, development and commercialization of new therapeutic drugs from cannabis In late June this year, epidilex (cannabidiol), an oral liquid preparation of cannabidiol, was approved by the FDA of the United States It is used in patients aged 2 and over to assist in the treatment of epilepsy related to Lennox Gastaut syndrome (LGS) and Dravet syndrome (DS) LGS and DS are two rare, severe and childhood epilepsy types, which are also the most difficult to treat This approval makes epidiolex the first high-purity, plant-derived cannabis diol (CBD) prescription drug preparation, as well as the first new antiepileptic drug (AED) At the beginning of this month, epidiolex officially went on sale in the United States The industry is very optimistic about the business prospects of epidiolex At the beginning of this year, Coriolis Vivian released a report predicting that if it goes public, the sales of epidilex in 2018 and 2019 will be 19 million US dollars and 266 million US dollars respectively, and it will reach 1191 million US dollars in 2022 Recently, GW published positive top line data from the second phase III clinical study of epidilex in the treatment of DS related epilepsy This also represents the fourth positive key study of epidiolex in the treatment of two kinds of intractable pediatric epilepsy (LGS, DS) Epidiolex's clinical development project currently includes four phase III randomized controlled LGS and DS studies and one open label extension study Data from three previously completed phase III studies have been published in the New England Journal of medicine and the lancet It is worth mentioning that epidiolex is the only FDA approved development project of plant-derived cannabinoids This is a randomized, double-blind, placebo-controlled phase III study involving 199 children and adolescents (2-18 years old) with refractory DS who received one or more antiepileptic drugs (AES) at the same time On average, these patients were taking three AEDs, and had previously tried and stopped taking four other AEDs, with an average age of 9 years (2-18 years), a median baseline seizure frequency of 12 per month, and a median baseline total seizure frequency of 35 per month In the study, these patients were randomly divided into three groups They received epidiolex 10mg / kg / day (n = 67), epidiolex 20mg / kg / day (n = 67) and placebo (n = 65) while receiving their current AED treatment The primary end point was the change in the frequency of TiC episodes relative to baseline during the 14 week treatment period (12 week maintenance period after 2-week drip) The results showed that the frequency of convulsions decreased by 49%, 46% and 27% compared with baseline in the epidilex 10mg / kg / day treatment group, epidilex 20mg / kg / day treatment group and placebo group respectively The data of the two doses of epidilex (10mg and 20mg) treatment group were statistically significant compared with the placebo group (P = 0.0299, P = 0.0095, respectively), reaching the main end point In addition, the two doses of epidilex (10mg and 20mg) treatment group also showed statistically significant improvement in all key secondary endpoints (including: total seizure reduction, ≥ 50% responder analysis, and total caregiver impression change scale [cgi-c]) compared with the placebo group During the treatment period, the proportion of patients whose total number of epileptic seizures decreased by more than 50% compared with the baseline was 49% in epidielex 20mg / kg / day treatment group, 44% in epidielex 10mg / kg / day treatment group and 26% in placebo group (P = 0.0069, P = 0.0332 respectively) In terms of safety, the safety data obtained in this study is consistent with the previous phase III clinical study, and with epidiolex's prescription information in the United States The most common adverse reactions (incidence ≥ 10% and more than placebo group) in patients treated with epidilex included: lethargy, anorexia, diarrhea, elevated transaminase, fatigue, discomfort, weakness, rash, insomnia, sleep disorders, poor sleep quality and infection The detailed results of the study will be presented at a future medical conference LGS is a kind of age-related cryptogenic or symptomatic generalized epilepsy syndrome, which is a type of age-dependent epilepsy encephalopathy It is characterized by early onset age, early childhood onset, various attack forms, intellectual development affected, and difficult treatment It is a serious type of epilepsy DS, also known as infantile severe myoclonic epilepsy, is a rare progressive epileptic encephalopathy mainly caused by genetic factors It has the characteristics of early onset age, various types of seizures, high frequency of seizures, serious intellectual damage, poor drug treatment effect, etc The prognosis of this disease is poor, and almost all children have cognitive impairment Original source: GW pharmaceuticals announcements second positive phase 3 pivotal trial for epidiolex? (cannabidiol) oral solution CV in patients with dravetsyndrome