The first new anti epileptic drug from cannabinoids! Epidiolex is a successful treatment for stage III epilepsy related to tuberous sclerosis (TSC)!

December 31, 2019 / BIOON / -- GW Pharma, a British pharmaceutical company, is a global leader in the research and development of plant-based cannabinoid treatment products It is committed to the discovery, development and commercialization of new treatment drugs from cannabis Recently, the company released the data of the phase III clinical study on the treatment of tuberous sclerosis (TSC) with epidilex (cannabidiol, CBD) oral liquid preparation The results showed that epidiolex significantly reduced refractory seizures associated with TSC and improved the overall condition of patients compared with placebo This is a randomized, double-blind, placebo-controlled trial conducted in 46 clinical centers in six countries, involving 224 TSC patients (aged 1-65 years) who were diagnosed as resistant to treatment (refractory) In the study, these patients were randomly assigned to receive epidilex 25mg / kg / day (n = 75), epidilex 50mg / kg / day (n = 73), placebo (n = 76) for 16 weeks (4-week titration, 12 week maintenance) The primary end point was the percentage change in the frequency of TSC related focal and systemic seizures compared to placebo during treatment The key secondary end points included: the proportion of patients with ≥ 50% reduction in seizures, the proportion of patients with ≥ 50% reduction in total seizure frequency (including focal sensation and epileptic spasm), and the overall impression of changes in the overall condition of subjects / caregivers (s / CGIC) The results showed that the main end point of the study was reached Compared with the placebo group, the frequency of TSC related epileptic seizures in the epidilex treatment group was significantly reduced: the relative baseline of the epidilex 25 mg / kg / day treatment group and epidilex 50 mg / kg / day treatment group was reduced by 49% and 48%, respectively, and that of the placebo group was reduced by 29% (P = 0.0009, P = 0.00118) Secondary end point data showed a 50% or greater reduction in seizures compared to placebo (36% in the 25mg / kg / day group, 40% in the 50mg / kg / day group, 22% in the placebo group, P = 0.0692 and P = 0.0245) In addition, 48% of the patients in the two doses of epidilex treatment group experienced a greater reduction in total seizure frequency (including focal sensation and epileptic spasm) compared with the placebo group, which was 27% (P = 0.0013 and P = 0.0018) According to the results of S / CGIC questionnaire, the improvement rates of epidilex 25mg / kg / day group and epidilex 50mg / kg / day group were 69%, 62% and 39% respectively (P = 0.0074 and P = 0.0580) Additional analysis showed that patients treated with epidilex experienced a greater reduction in complex focal seizures than those treated with placebo (52% and 50% respectively in the 25 mg / kg / day and 50 mg / kg / day treatment groups, 32% in the placebo group, P = 0.0076 and P = 0.0116) The safety profile observed in this study is basically consistent with the previous results The incidence of adverse events (AE) was 93% in the 25mg / kg / day group, 100% in the 50mg / kg / day group and 95% in the placebo group Both doses have acceptable safety, and adverse events of 25 mg / kg / day are less than 50 mg / kg / day The most common adverse reactions were diarrhea, loss of appetite and drowsiness In the 25 mg / kg / day group, 50 mg / kg / day group and placebo group, 5 cases, 10 cases and 2 cases respectively stopped treatment due to adverse events In addition, 13% and 25% of the patients in the 25 mg / kg / day and 50 mg / kg / day groups showed elevated liver enzymes, and 79% of them were also taking the antiepileptic drug sodium valproate ALT / AST was relieved in all patients No cases of Hy's law were observed and no deaths were reported "Epilepsy is the most common neurological feature of TSC," said Dr Elizabeth Thiele, investigator of the study, Professor of Neurology at Harvard Medical School, Professor of Neurology at Harvard Medical School, director of pediatric epilepsy at Massachusetts General Hospital, and director of Carol and James herscot tuberous sclerosis center TSC is a rare, serious and childhood disease Up to two-thirds of the patients will experience drug refractory epilepsy In order to solve the epilepsy related to TSC, there is a great demand for new treatment methods These positive results suggest that patients may benefit from the treatment of epidilex, a high-purity CBD preparation " Tuberous sclerosis (TSC) is a rare genetic disease, affecting about 50000 people in the United States and nearly 1 million people in the world Globally, at least two TSC babies are born every day, with an estimated one in 6000 births The main cause of hereditary epilepsy is the growth of benign tumors in important organs of the body, including brain, skin, heart, eyes, kidney and lung TSC usually occurs in the first year of life, manifested as focal epilepsy or infantile spasm, and is associated with an increased risk of autism and intellectual disability The severity of the condition can vary widely In some children, the disease is very mild, while others may have life-threatening complications About 85% of TSC patients have epilepsy, which may develop into drug refractory More than 60% of TSC patients can't control epilepsy by standard treatment, such as antiepileptic drugs, epileptic surgery, ketogenic diet or vagus nerve stimulation, compared with 30-40% of epilepsy patients without TSC are resistant Epidyolex (European trade name: epidyolex) is an oral, high-purity CBD extract liquid preparation CBD is a non spiritual component from cannabis plant, which has a variety of pharmacological effects on the nervous system A large number of studies have shown that CBD has obvious antiepileptic and anticonvulsant activities, with fewer side effects than existing antiepileptic drugs In the United States, epidilex oral liquid preparation was approved by FDA in June 2018 for use in patients aged 2 and over to assist in the treatment of epilepsy related to Lennox Gastaut syndrome (LGS) and Dravet syndrome (DS) In the EU, epidyolex was approved for the same indications in June 2019 LGS and DS are two rare, severe and childhood epilepsy types, which are also the most difficult to treat In the United States, FDA has granted epidilex orphan status in the treatment of LGS and DS In addition, FDA has granted epidiolex a fast track status in the treatment of DS In Europe, epidyolex has also been granted orphan status by EMA to treat LGS and DS Epidilex / epidyolex is the first plant-derived cannabinoid drug approved by the United States and Europe for the treatment of epilepsy, and also the first new antiepileptic drug (AED) The industry is very optimistic about the commercial prospect of the drug The company had previously forecast sales of $1 billion 200 million in 2022 At present, GW pharmaceutical company is developing epidilex / epidyolex for the treatment of other rare diseases including TSC and Rett syndrome GW has launched sativex, the world's first herbal cannabinoid prescription drug, which has been approved in many countries outside the United States for the treatment of multiple sclerosis spasm; the company is advancing a later stage project of sativex to seek FDA approval The company has a range of cannabinoid candidates in its pipeline, including compounds for epilepsy, autism, glioblastoma and schizophrenia Original source: Greenwich Biosciences, a GW pharmaceuticals company, presentations new data for epidiolex? (cannabidiol) in patients with tuborous Sciences Complex (TSC) at the American Epilepsy Society annual meeting