GW Pharmaceuticals has also received approval for the formulation in the European Union under the name Epydiolex.
The company stated earlier this month that Epydiolex was under review at the European Medicines Agency for use in treating TSC.
The recent approval applies to TSC patients one year of age and older.
The approval also extends the age range of Lennox-Gastaut syndrome and Dravet syndrome patients to one year of age and older.
Tuberous Sclerosis Complex
TSC, one of the most common causes of epilepsy, is a disease that manifests as tumors in vital organs such as the skin, heart, brain, eyes, lungs, and kidneys.
While some TSC patients may experience mild symptoms, others may experience life-threatening complications.
Epilepsy exists in approximately 85 percent of TSC patients and may become resistant to medication.
GW Pharmaceuticals CEO Justin Gover says FDA approval of Epidiolex for TSC seizures is exciting news.
“Since Epidiolex is already available to patients by physician’s prescription, patients with TSC can immediately access the medication,” says Gover. “This label expansion, including the expansion of the age range in all approved indications, further demonstrates that the FDA process can continue to enable broader patient access to appropriately tested regulatory approved cannabinoid medicines. It also provides hope for these patients and their families and is yet another important milestone for Epidiolex as a first-in-class antiepileptic drug.”
A Promising Treatment
FDA approval includes a recommended dose of 25 mg/kg per day for TSC patients, based on Phase 3 safety and efficacy study data.
GW Pharmaceuticals reports that the study met its primary endpoint—showing that Epidiolex could reduce seizure frequency compared to a placebo.
The result was a seizure reduction in 48 percent of patients who took the recommended Epidiolex dose, compared to 24 with a placebo.
GW Pharmaceuticals says the safety profile observed in the study was generally comparable to that of prior Epidiolex studies.
Director of the Herscot Center for Tuberous Sclerosis Complex at Massachusetts General Hospital Elizabeth Thiele, M.D., Ph.D. says that positive trial results indicate that Epidiolex may become an important patient treatment option.
“It is a new tool in the toolbox for physicians and could meet a significant unmet need,” says Thiele. “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.”
According to the TSC Alliance, TSC affects approximately 50,000 people in the United States and nearly one million worldwide.
GW Pharmaceuticals says at least two children are born with TSC each day, with an estimated prevalence of one in 6,000 newborns.
Tuberous Sclerosis Alliance President and CEO Kari Luther Rosbeck says FDA approval is a tremendous step forward.
“One of the most challenging and frustrating aspects of TSC are seizures that cannot be effectively controlled by existing medications,” says Rosbeck. “New treatment options are desperately needed, and this approval adds another option for those impacted by this difficult disease.”