Ongoing study demonstrates Brineura's durable treatment benefit

An ongoing open-label extension study treating patients with brain disease drug Brineura (cerliponase alfa) continued to show a reduced rate of decline compared to a natural history cohort of CLN2 disease for three years as measured by the CLN2 Clinical Rating Scale, according to US biotech firm BioMarin Pharmaceutical (Nasdaq: BMRN), whose share gained 1.56% to $94.69 following the news on Friday.

The data presented show a durability of treatment effect in the primary efficacy endpoint where response to treatment was seen in 19 of 23 or 83% of treated patients after three years. A response is defined as the absence of an unreversed two-point decline in the motor-language (ML) scale or a score of 0, outcomes that represent clear and substantial decline in motor and language function. Natural history patients were 12 times more likely on average to have experienced an unreversed two-point decline in ML score than treated patients at three years. After three years on treatment with Brineura, treated patients' ML scores were on average 3.8 points better than natural history. After two years on treatment with Brineura, treated patients' ML scores were on average 3.3 points better than natural history.

Already approved in USA and Europe