BioMarin says Pegvaliase application remains on track
BioMarin said its Pegvaliase biologics license application remains on track for FDA action during the first half of 2018. The company plans to submit a Marketing Authorization Application to the European Medicines Agency in Q1 of 2018. BioMarin also provided an update on its open-label Phase 2 study of vosoritide, an analog of C-type Natriuretic Peptide, in children with achondroplasia, the most common form of disproportionate short stature in humans. "Vosoritide for achondroplasia demonstrates sustained increase in average growth velocity over 30 months of treatment in 10 children," the company said. It added, "Over this period of time, patients have experienced mean absolute growth increase of approximately 4 cm over what their baseline growth velocity would have predicted. The sustained increase in annualized growth velocity was accompanied by sustained improvements over time in height compared to age- and gender-matched unaffected children as measure by z-scores. In addition, treatment with vosoritide shows continued improvement over time in proportionality as measured by a ratio of the upper and lower body measurements or U/L ratio."