FDA Approves EPIDIOLEX (cannabidiol) Oral Solution to Treat Seizures Associated with Tuberous Sclerosis Complex - Seite 2

“Based on previous positive trial results in TSC patients, EPIDIOLEX may become an important treatment option for patients. It is a new tool in the toolbox for physicians and could meet a significant unmet need," said Elizabeth Thiele, M.D., Ph.D., Director of the Herscot Center for Tuberous Sclerosis Complex at Massachusetts General Hospital, Professor of Neurology at Harvard Medical School and clinical investigator. “Nearly two-thirds of individuals with TSC develop treatment-resistant epilepsy and there is a need for new options that may benefit these patients who often try and fail existing treatments.”

The Company anticipates most payers will quickly update their EPIDIOLEX coverage policies to include TSC at access levels similar to that of LGS and Dravet syndrome.

“FDA approval of EPIDIOLEX in TSC is a tremendous step forward and our community applauds this positive development,” said Kari Luther Rosbeck, President and CEO of the Tuberous Sclerosis Alliance. “One of the most challenging and frustrating aspects of TSC are seizures that cannot be effectively controlled by existing medications. New treatment options are desperately needed, and this approval adds another option for those impacted by this difficult disease.”

FDA approval includes a recommended maintenance dose of 25 mg/kg/day for TSC patients, which is supported by data from a Phase 3 safety and efficacy study evaluating 25 mg/kg/day of EPIDIOLEX. The study met its primary endpoint, which was the reduction in seizure frequency compared to baseline of Epidiolex vs placebo, with seizure reduction of 48 percent in patients taking Epidiolex 25 mg/kg/day compared with 24 percent for placebo (p<0.01).  All key secondary endpoints were supportive of the effects on the primary endpoint. The most common adverse events in those receiving EPIDIOLEX in the study (≥10 percent and greater than placebo) included diarrhea; transaminase elevations; decreased appetite; somnolence; pyrexia; and vomiting. The safety profile observed in this study was generally comparable to that observed in prior studies of Epidiolex.

About Tuberous Sclerosis Complex (TSC)
Tuberous sclerosis complex (TSC) is a rare genetic condition that affects approximately 50,000 individuals in the U.S. and nearly one million people worldwide.¹ At least two children born each day will develop TSC, with an estimated prevalence of one in 6,000 newborns.⁵ The condition causes mostly benign tumors to grow in vital organs of the body including the brain, skin, heart, eyes, kidneys and lungs ² and is a leading cause of genetic epilepsy.³ People with TSC may experience a variety of seizure types. One of the most common is infantile spasms that typically present in the first year of life; focal (or partial) seizures are also very common.⁴ TSC is associated with an increased risk of autism and intellectual disability⁵ and the severity of the condition can vary widely. In some children the disease is very mild, while others may experience life-threatening complications.⁶ Epilepsy is present in about 85 percent of patients with TSC and may progress to become intractable to medication.^1,2,3 More than 60 percent of individuals with TSC do not achieve seizure control⁴ with standard treatments such as antiepileptic drugs, epilepsy surgery, ketogenic diet, or vagus nerve stimulation² compared to 30-40 percent of individuals with epilepsy who do not have TSC who are drug resistant.^6,7