Epidiolex delivers positive results for a third rare condition called TSC

Epidiolex reports positive results for its drug when treating tuberous sclerosis complex (TSC) a rare genetic condition. The condition causes mostly benign tumors to grow in vital organs of the body including the brain, skin, heart, eyes, kidneys and lungs and is a leading cause of genetic epilepsy.

Epidiolex was the leading medical marijuana drug originally produced by GW Pharmaceuticals to treat the rare pediatric epilepsy diseases – Dravat Syndrome and Lennox Gastaut. GW Pharmaceuticals was later acquired by Jazz Pharmaceuticals (NASDAQ: JAZZ). Epidiolex was the first medical non-synthetic marijuana drug to go through the highly regulated FDA study trials. It has been instrumental in reducing the stigma of medical marijuana by demonstrating significant reductions in seizures for children with these diseases.

Real-world data

“Our real-world data presentations at AES 2024, including novel findings from the BECOME-LTC, BECOME-TSC and EpiCom studies, demonstrate the meaningful impact of Epidiolex in the treatment of patients with rare epilepsies,” said Sarah Akerman, MD, head of neuroscience global medical and scientific affairs of Jazz Pharmaceuticals. “These findings increase our understanding of Epidiolex’s benefits beyond seizure control, addressing unmet needs across a range of epilepsy syndromes for people living with rare epilepsies and demonstrating reproducibility and consistency of effect across different populations.” BECOME-TSC refers to BEhavior, COgnition, and More with the Epidiolex caregiver survey.

The company will present the results at the American Epilepsy Society (AES) 2024 Annual Meeting, which will be held this week in Los Angeles, California. The company reported some of the findings as follows:

• A three-month study demonstrated improvements after an Epidiolex treatment in the severity of behavioral problems in patients with TSC as reported by the TAND Self-Report Quantified Checklist and Aberrant Behavior Checklist.

• Two updated analyses showed that, of 55 caregivers who completed the survey, 89% planned to continue Epidiolex treatment for their loved one. The most important reasons for continuing Epidiolex included seizure and non-seizure benefits such as reduced seizure frequency and severity/duration and TAND-related improvements in cognition and language/communication.

• Results from the BECOME-LTC (BEhavior, COgnition, and More with Epidiolex in the Long-Term Care Setting) survey found that, among 102 nurses surveyed, 85% reported a reduction in the overall frequency of any seizure type after Epidiolexinitiation, with 49% reporting a greater than 50% reduction.

• Results from CARE-EpiC (Caregiver Analysis of Real-world Epidiolex in Epilepsy Context), a cross-sectional caregiver survey, demonstrated reduced caregivers’ need for additional support of their dependents’ physical, emotional, and behavioral care after Epidiolex initiation and characterized improvements in their dependents’ well-being as well as caregivers’ experiences.

• In the TSC group, Epidiolex was associated with a median reduction from baseline of 51%–87% in focal seizures and 44%–87% in total seizures. In the non-TSC group, Epidiolex was also associated with a median reduction from baseline of 46%–75% in focal and 46%–74% in total seizures.

Epidiolex success

Green Market Report wrote in November that Epidiolex, accounted for $251.5 million in revenue for the company’s third quarter, up from $213.7 million the same quarter a year ago. For the past nine months ending in September, Epidiolex has generated $697 million in sales – an increase of 15% over the previous year. Epidiolex, which is now available in 35 countries, was second in sales only to Xywav, Jazz’s sleep aid drug, which sold $388.4 million in the quarter.

The company has been facing patent lawsuits from other medical marijuana companies including Teva Pharmaceuticals, Inc. and Ascent Pharmaceuticals. Some of the original claims to create a generic version of the drug dating back to 2022 were dismissed but others were allowed to move forward. Jazz managed to reach settlements with some parties, but then new cases were filed. A trial is scheduled for September 2025.

Separately, On November 20, 2024, Jazz Pharmaceuticals announced that the FDA accelerated approval of its Ziihera (zanidatamab-hrii) 50mg/mL for injection for IV for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC). The FDA granted accelerated approval for this indication based on overall response rate and duration of response.