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Who Is GW?
GW Pharmaceuticals was founded in 1998, with operations in both the US and the UK. GW is dedicated to developing plant-derived cannabinoid therapeutics. Their lead product is Epidiolex. It is pure cannabidiol extract for rare, early-onset, drug-resistant severe forms of epilepsy. GW also developed the world’s first cannabis based prescription medicine, Sativex, intended for treatment of spasticity due to multiple sclerosis.
The company has a pipeline of clinical stage cannabinoid products focused on neurological conditions: Epidiolex for Dravet Syndrome, Lennox Gastaut Syndrome, and Tuberous Sclerosis; CBDV (GWP42006) for Epilepsy and Autism Spectrum Disorders; GWP42003 (IV) for Neonatal Hypoxic-Ischemic Encephalopathy; GWP42002/ GWP42003 for Glioblastoma; and GWP42003 for Schizophrenia.
GW’s primary goal is to “maintain a world leading position in the field of cannabinoid science and in the research, development and commercialization of cannabinoid molecules as novel prescription pharmaceutical therapeutic candidates.” GW’s Chief Executive Officer, Justin Gover, defines the company’s values here in their mission statement.
Our primary purpose is to make a positive and meaningful difference in the lives of patients and their caregivers. All of our actions and decisions tie back to this most important and simple fact. Equally, we want our employees to understand and trust that GW Pharmaceuticals and its US subsidiary, Greenwich Biosciences, is committed to doing the right thing. So, we have made it clear what we expect from all of our colleagues.
What Is Epidiolex?
Epidiolex is GW’s lead cannabinoid product candidate. It is an oral solution of pure plant derived cannabidiol (CBD). The development of epidiolex includes two distinct programs. The first is a Food and Drug Administration (FDA) authorized clinical trials program. They are currently conducting an ongoing series of clinical trials intended to assure the safety and efficacy of Epidiolex, which is necessary to be approved as a prescription medication.
In the trials, eligible patients are randomly and blindly assigned either Epidiolex or a placebo. These are added to their current treatment and evaluated over a span of time. Epidiolex is current undergoing two phase 3 trials in Dravet syndrome, two in LGS, one in TSC, and one in IS. The first of the two trials showed significant reduction in seizure types for patients taking Epidiolex compared to those taking the placebo.
The second program is a FDA-authorized, independent Physician-led program or Expanded Access (Compassionate Use program). Within a compassionate use program, the FDA may authorize expanded access to investigational drugs to treat patients with serious or immediately life-threatening diseases or conditions that lack medical alternatives. This is done through Investigational New Drug (IND) applications through the FDA.
The FDA has granted individual patient emergency INDs to physicians and state programs to treat patients suffering from intractable (uncontrollable) epilepsy. Results from 261 INDs patients receiving Epidiolex showed promising signals in reducing seizures.
What Is Epidiolex For?
Epidiolex is primary focused on severe, orphan, early-onset, treatment-resistant epilepsy syndromes. These severe forms of epilepsy would include Dravet syndrome, Lennox-Gastaut syndrome (LGS), Tuberous Sclerosis Complex (TSC) and Infantile Spasms (IS).
Dravet syndrome is a genetic dysfunction of the brain. It is rare and begins in the first year of life. It is lifelong and causes most children to develop some type of developmental disability.
Lennox-Gastaut Syndrome is a type of epilepsy with multiple different types of seizures. A child’s intellectual development is usually impaired. In approximately a quarter of children, no cause can be confirmed. The patient’s seizures don’t normally respond to conventional medications. LGS accounts for only 2-5% of childhood epilepsies. LGS normally lasts through childhood, adolescence, to adulthood.
Tuberous Sclerosis Complex (TSC) is a rare genetic disease that causes benign tumors to grow in the brain and on other organs such as the kidneys, heart, eyes, lunds, and skin. It often impacts the central nervous system and results in certain symptoms, including seizures, developmental delay, behavioral problems, skin abnormalities, and kidney disease.
Infantile Spasms are a specific type of seizure seen in West Syndrome, known to impact infants and children. West Syndrome is defined by infantile spasms, developmental regression, and a specific pattern on electroencephalography (EEG) testing called hypsarrhythmia (chaotic brain waves). The spasms begin usually in the first year of life, most often between four to eight months. The spasms normally appear as a sudden bending forward of the body with stiffening of the arms and legs. Some children arch their backs and extend their arms and legs. Spasms normally occur when awakening or after feeding. They often include up to 100 spasms at a time.
Organizations that provide help, information, and support to people living with epilepsy.