Phase III study of BMN 111 shows positive results in achondroplasia.- BioMarin Pharma

BioMarin Pharmaceutical reported positive final results from its randomized, double-blind, placebo-controlled Phase III study evaluating the efficacy and safety of BMN 111 (vosoritide) in 121 children aged 5 to 14 with achondroplasia. The placebo-adjusted change from baseline in growth velocity after one year of treatment with vosoritide, the primary endpoint, was 1.6 cm/yr (p<0.0001). vosoritide is an investigational once daily injection analog of c-type natriuretic peptide cnp. achondroplasia is the most common form of disproportionate short stature. the results were consistent across the broad patient population studied.>

Vosoritide, administered at 15ug/kg/day in this Phase III randomized, double-blinded placebo-controlled study over one year, was generally well tolerated. The majority of adverse events (AEs) were mild and no serious adverse events were reported as study drug-related. Injection site reactions were the most common drug-related AEs, and all were transient. No clinically significant blood pressure decreases or new safety findings were observed. Based on these results, the Company plans to meet with health authorities in the first half of 2020 to discuss plans for submitting marketing applications.

Comment: Achondroplasia, the most common form of disproportionate short stature in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face and base of the skull. This condition is caused by a mutation in the fibroblast growth factor receptor 3 gene (FGFR3), a negative regulator of bone growth.