ARTICLE | Clinical News

Ganaxolone: Additional Phase II data

October 24, 2016 7:00 AM UTC

Additional data from 11 female patients ages 4-15 with protocadherin 19 (PCDH19) epilepsy and uncontrolled seizures despite antiepileptic pharmacotherapy in an open-label, U.S. and Italian Phase II trial showed that adjunct treatment with up to 63 mg/kg oral ganaxolone once daily for 26 weeks reduced seizure frequency per 28 days compared to baseline in 7 (64%) patients, including 4 patients with a >50% reduction from baseline. PCDH19 female pediatric epilepsy is a rare epileptic syndrome caused by a mutation on the PCDH19 gene. On secondary endpoints, ganaxolone increased seizure-free days in 73% of patients and led to Clinical Global Impression-Improvement (CGI-I) scores of very much improved, much improved or minimally improved in 73% of patients. The product was generally well tolerated with somnolence, headache, seizure and fatigue reported as the most common treatment-related adverse events...