Jazz Pharma is breaking new ground with its cannabidiol epilepsy treatment Epidiolex, taking the drug into a Phase III study in children and adolescents.
Jazz Pharma is breaking new ground with its cannabidiol epilepsy treatment Epidiolex, taking the drug into a Phase III study in children and adolescents.
The study will include children and adolescents living with developmental and epileptic encephalopathy, known as epilepsy with myoclonic-atonic seizures (EMAS).
In 2018, Epidiolex, developed by GW Pharmaceuticals, became the first cannabis-based drug approved by the FDA and required a reclassification by the U.S. Drug Enforcement Agency prior to commercialization. Epidiolex is derived from cannabidiol (CBD), a chemical component of the Cannabis sativa plant, more commonly known as marijuana. However, CBD does not cause the typical “high” associated with marijuana, as it does not include tetrahydrocannabinol (THC), the plant’s main psychoactive compound.
Epidiolex was approved by the FDA for the treatment of seizures associated with two rare forms of epilepsy: Lennox-Gastaut syndrome (LGS) and Dravet syndrome. Jazz Pharma acquired GW last year for $7.2 billion.
Now, Jazz is aiming Epidiolex at EMAS, which is also known as Doose syndrome. EMAS typically begins in early childhood. It is a developmental and epileptic encephalopathy that is fairly uncommon, accounting for 1% to 2% of all childhood-onset epilepsies, according to the Epilepsy Foundation. The disease tends to occur in boys more often than girls and is associated with different types of seizures, including drop seizures, tonic seizures and myoclonic seizures, among others.
Jazz noted that seizures in pediatric EMAS patients can be difficult to treat, and some of the children do not respond well to available medications. Currently available treatments for Doose syndrome include the anticonvulsants divalproex sodium, zonisamide, felbamate and others. According to the Epilepsy Foundation, some anticonvulsants can actually worsen the symptoms of EMAS patients.
Rob Iannone, executive vice president, global head of research and development of Jazz Pharmaceuticals, pointed out that there are numerous treatment-resistant epilepsy syndromes. Because of that, epileptologists, neurologists who specialize in epilepsy treatment, typically look for efficacy by seizure type, he said. Iannone further noted that most of those types have no syndrome-specific approved treatment.
“An EMAS indication would provide support for the use of Epidiolex in a fourth indication of a distinct, generalized seizure type, myoclonic-atonic seizures,” Iannone said in a statement. “Jazz is committed to continuing to generate clinical study data and real-world evidence to further support the utility of the company’s cannabidiol across a broad range of difficult-to-treat seizure types.”
The Phase III trial will assess Epidiolex over a 14-week treatment period in a randomized, double-blind, placebo-controlled study. The trial will consist of two parts and enroll pediatric and adolescent patients from the ages of 1 to 18.
The first portion of the study will assess the efficacy and safety of cannabidiol compared to placebo as an adjunctive treatment for children and adolescents with myoclonic-atonic seizures. When that portion of the study is completed, the patients will have an option to continue in a 54-week open-label extension trial, Jazz said.
Cannabis-based products are being assessed for multiple neurological indications. In addition to Epidiolex, Jazz is also developing Sativex, a cannabis-based drug approved in the United Kingdom for the treatment of multiple sclerosis. However, Sativex failed to reach key endpoints in a Phase III study in June.
Other companies are also developing cannabis-based products, including Cara Therapeutics, Corbus Pharmaceuticals and Insys Therapeutics. A recent study published in the Journal of Clinical Psychiatry showed that cannabis-based treatments can significantly reduce anxiety, with cannabidiol reducing anxiety by 50% in young people aged 12-25 who have not responded well to other treatments. Some researchers are also assessing cannabidiol assets against the inflammation associated with COVID-19 infections.
