BioMarin Drug Meets Endpoint and Will Seek FDA Approval

The company now plans to submit its marketing applications in the first quarter of 2013. The endpoint target was a change in six-minute walk distance compared with placebo at 24 weeks in subjects receiving weekly infusions of GALNS. The study was said to be a randomized, double-blind, placebo-controlled study, and it evaluated two doses of GALNS for the treatment of patients with the rare lysosomal storage disorder Mucopolysaccharidosis Type IVa.

Biomarin showed that patients dosed with GALNS at 2 mg/kg every other week did not show a meaningful or statistically significant change when evaluating the baseline compared to the placebo.

Shares are surging by more than 28% to $48.20, and this will mark a new 52-week high as the prior range was $30.06 to $44.18.

We would caution on one thing here. While MPS IVA is one of about 40 types of related genetic disorders, the overall population for this drug target is very small. BioMarin said:

The rate of incidence of MPS IVA is as yet unconfirmed and varies among different populations but estimates vary between 1 in 200,000 live births and 1 in 250,000 live births. The estimated prevalence is between 1,000 and 1,500 patients in the U.S., EU and Japan and between 1,500 to 2,000 patients in the rest of the world for a total of 2,500 to 3,000 patients.

That may be too small a market to add significantly to a company that already was worth $4.6 billion before this pop on the news.

JON C. OGG