The first plant-derived cannabinoid new anti-epileptic drug! FDA approves Epidiolex: Treatment of nodule sclerosis (TSC)-related epilepsy.

!--webeditor: "page title" -- GW Pharma, a UK pharmaceutical company, is a global leader in the science, development and commercialization of cannabinoid prescription drugs.
, the company and its U.S. subsidiary Greenwich Biosciences announced that the U.S. Food and Drug Administration (FDA) has approved a new indication of the cannabinoid drug Epidiolex (cannabidiol, cannabidiol, CBD) oral liquid formulation for the treatment of seizures associated with nodule sclerosis (TSC) in patients with 1 year of age.
the indication sist ame is approved through the priority review process.
in Europe, applications for the drug (product name: Epidyolex) for the same indications are being reviewed by the European Medicines Agency (EMA).
Epidiolex is the second FDA-approved drug to treat TSC-related seizures.
Novoolimus is the first drug to be approved for TSC-related seizures, which was approved by the European Union in January 2017 and April 2018 (product name: Votubia) and the United States (product name: Afinitor DISPERZ) for the treatment of some of the TSC-related seizures in children and adults.
Epidyolex/Epidiolex is the first plant-derived cannabinoid drug approved in the United States and Europe to treat epilepsy, and the first new anti-epileptic drug (AED).
in the United States and the European Union, the drug was approved for sale in June 2018 and June 2019, respectively.
the industry is very optimistic about the commercial prospects of the drug.
Corey,o'n s forecast that sales would reach $1.2 billion in 2022.
approval is the third regulatory indication that Epidiolex has received. Prior to
, the drug had been approved in the United States and the European Union for use in children and adults in 2 years of age and adults, to assist in the treatment of epilepsy associated with Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS).
in the United States and the European Union, Epidyolex/Epidiolex has been granted the right to treat DS, LGS, TSC-related seizures, each of which is a rare, severe, childhood-related type of drug refractive epilepsy.
TSC is a rare, severe, childhood-borne genetic disorder.
epilepsy is the most common neurological characteristic of TSC, as many as two-thirds of TSC patients experience drug-incurable seizures, there is a significant need for new treatments in the resolution of TSC-related seizures.
data from Phase III clinical studies show that Epidiolex significantly reduces TSC-related refractory seizures (including pontoon and systemic) compared to placebo and improves the overall condition of patients.
with the approval of this new indication, Epidiolex will provide an important treatment option for the TSC patient population.
nodule sclerosis syndrome -TSC (photo source: childchildyfoundation.org) this approval is based on the results of a randomized, double-blind, placebo-controlled Phase III clinical study.
the study included 224 patients (aged 1-65) who were diagnosed with TSC patients who were resistant to treatment (difficult to treat), who were randomly assigned to receive Epidiolex 25mg/kg/day (n-75), Epidiolex 50mg/kg/day (n-73), placebo (n-76), and treatment for 16 weeks (4 weeks of regular, 12 weeks of maintenance). the main endpoint of
was the percentage change of the ratio of The Frequency of Epidiolex to placebo during treatment compared to the rate of TSC-related episodes and systemic seizures relative to the baseline.
key secondary endpoints include: a reduction in the proportion of patients with seizures of more than 50%, a reduction in the overall frequency of seizures (including teratolic sensations and epilepsy spasms), and an overall impression of changes in the overall condition of the subject/caregiver (S/CGIC).
results showed that the study reached the primary endpoint, compared to the placebo group, the Epidiolex treatment group TSC-related seizure frequency significantly reduced: Epidiolex 25 mg/kg/day treatment group, 50 mg/kg/day treatment group decreased by 49%, 48%, and placebo group decreased by 27% (p-0.0009, p-0.00118).
the results of all critical secondary endpoints support the impact on the primary endpoint.
specifically: (2) a higher proportion of patients in the Epidiolex treatment group had a 50% or larger reduction in seizures (36% in the 25mg/kg/day group, 40% in the 50mg/kg/day group, 22% in the placebo group, and p-0.0692 and p-0.0245).
(2) had a significant decrease in the total seizure frequency (including pentorial sensation strains) in 48% of patients in both doses of Epidiolex treatment group compared to the placebo group(2) and 27% in the placebo group (p-0.0013 and p-0.0018).
(3) reported improvements in the overall status of Epidiolex 25mg/kg/day group, Epidiolex 50mg/kg/day group, 69%, 62%, and placebo group by 39% (p-0.0074 and p.0580), respectively, based on the overall impression of patients/caregivers (S/CGIC) questionnaire.
(4) additional analysis showed that patients treated with Epidiolex experienced a significant reduction in combined episode epilepsy compared to placebo patients (25 mg/kg/day treatment group, 50 mg/kg/day treatment group, 52%, 50%, placebo group 32%, p-0.0076 and p-0.0116).
the security profile observed in the study was consistent with previous research findings and no new security risks were found. the incidence of adverse events (AE) in
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, with adverse events of 25 mg/kg/day being less than 50 mg/kg/day. the most common adverse reactions
were diarrhea, loss of appetite and drowsiness.
!--/ewebeditor:!--.s.: !--." ."-nosy-sclerosis (TSC) is a rare genetic disease that affects about 50,000 people in the United States and nearly 1 million worldwide.
globally, at least two TSC babies are born every day, an estimated one in every 6,000 newborns.
the disease is mainly caused by benign tumors to grow in vital organs of the body, including the brain, skin, heart, eyes, kidneys and lungs, which are the leading causes of hereditary epilepsy.
TSC usually occurs in the first year of life, manifested as connoissal epilepsy or infantile spasms, and is associated with an increased risk of autism and intellectual disability. the severity of the
may vary widely.
In some children, the disease is very mild, while others may develop life-threatening complications.
about 85% of TSC patients have epilepsy and may develop into a drug that is difficult to treat.
more than 60% of TSC patients are unable to control seizures with standard treatments such as anti-epileptic drugs, epilepsy surgery, ketogenic diet or vagus nerve stimulation, compared with 30-40% of patients without TSC.
Epidyolex/Epidiolex is an oral, high-purity CBD extract liquid preparation, CBD is a non-psychoactive 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 anticonvulsive activity, with fewer side effects than existing anti-epileptic drugs.
Epidyolex/Epidiolex is the first plant-derived cannabinoid drug approved in the United States and Europe to treat epilepsy, and the first new anti-epileptic drug (AED).
in the United States, Epidiolex was approved in June 2018.
in the European Union, Epidyolex was approved in June 2019.
the industry is very optimistic about the commercial prospects of the drug.
Corey,o'n s forecast that sales would reach $1.2 billion in 2022.
currently, GW Pharmaceuticals is developing Epidiolex/Epidyolex to treat other rare diseases, including Rett syndrome.
GW, which has launched the world's first plant-sourceed prescription cannabicide drug Sativex (nabiximols), which has been approved outside the United States to treat spasms of multiple sclerosis, is pushing a late-stage project for Sativex to seek FDA approval.
the company has a range of cannabinoid candidates in its pipeline, including compounds for the treatment of epilepsy, autism, glioblastoma, and schizophrenia.
original source: FDA approves New Good for Drug Penting an Active Ingredient Derived from Cannabis to Treat Treats in Rare Genetic Disease !--/ewebeditor: page.