Brineura (cerliponase alfa) seems to be showing positive results in the management of children with CLN2 Disease. The reduced rate of decline is not comparable to a natural history cohort of CLN2 disease. This is according to BioMarin Pharmaceutical Ltd and as per the results on the ongoing open-label extension study. BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) has been surging consistently to new highs. The stock gained 0.55% in the last trading session here is the reason why the stock is gaining new heights.
Of the 23patients treated after three years, 19 of them responded with the primary efficacy endpoint effect, which indicated the durability of the treatment. The patient’s motor-language (ML) scores were on average 3.8 points. This was incredible compared to the patients with a natural history or those who had used Brineura for two years.
Brineura is the first to have the FDA’s approval to treat children with CLN2 disease
CLN2 is a form of Batten disease, which affects the brain in children. The victims experience seizures between the ages of two and four years old and the result is usually language development delay. The diseases progress at a very rapid rate and the affected children are likely to lose the ability to walk. In the later stages, it becomes difficult to attend to everyday activities like feeding.
However, the approval of Brineura by the FDA bring some hope the children suffering from this destructive disease. Apparently, this is the first treatment to obtain the agency’s approval, and it is such a milestone for the company.
According to Angela Schulz, the lead investigator, “Every day, week, month and year of maintaining clinical function, including language and mobility, is critical to children with CLN2 disease and their families.”
But the sales of Brineura remains slow
The incidence of CLN2 disease is more often than not one in 200,000 children even though many remain undiagnosed. This means the number of CLN2 disease patients has remained relatively low. It also explains why the launch and the sales of Brineura have remained low and just under $11 million during the quarter. Nonetheless, given that there are no other drugs available for the disease, there is optimism that things could get better.