Regulatory Decision Summary for Brineura

The efficacy and safety of Brineura were assessed over 96 weeks in an open-label clinical study and an ongoing long-term extension study in patients with CLN2 disease compared to an untreated historical control group. Twenty-three patients, aged 3 to 8 years, received Brineura at 300 mg every other week to a maximum of 145 weeks.

The primary efficacy endpoint was the proportion of patients with an absence of an unreversed two-point decline on a disease-specific clinical rating scale for the motor and language functions. Efficacy was demonstrated at Week 48 of the open-label study where 87% of the patients met the primary endpoint. The proportion of patients with no decline at both Week 48 and Week 96 (Week 48 of the long-term extension study) was 83% and showed durability of treatment effect.

The major risks were severe or life-threatening device-related infections and meningitis. The most common adverse events (AEs) were pyrexia (71%), vomiting (63%) and seizure (58%). The most common serious AE was hypersensitivity (29%). There were no deaths during the clinical development program. Given the poor prognosis of this disease, the safety profile of Brineura is considered acceptable with the appropriate labelling and post-market monitoring that has been put in place through the Brineura Risk Management Plan.

Overall, the benefits of Brineura outweigh its risks for the indication sought. The benefit/risk profile of Brineura is favourable in the target patient population.

For more information on Health Canadas decision, please view the Summary Basis of Decision.