BioMarin Pharmaceutical Stock Rises On Favorable Phase 2 Study Of Vosoritide

BioMarin Pharmaceutical Inc. BMRN shares rose 4.7 percent after the company reported the findings from the Phase 2 trial offered evidence of durability of effect consistent with the earlier presented six-month data for those patients. Incidentally that demonstrated an annualized 50 percent or 2.0cm/year increase in mean annualized growth velocity.

The company stated that additionally, the 6-month data for 12 patients who were initiated on a lower dose and switched to 15 µg/kg/day demonstrated an increase of 65% or 2.3 cm/year in mean annualized growth velocity from baseline (p-value = 0.002).

BioMarin's Chief Medical Officer, Hank Funchs, said that "We're encouraged by the consistency of the data from six to 12 months in both safety and efficacy, and plan to initiate a Phase 3 study by the end of the year. By addressing the root cause of achondroplasia with vosoritide treatment and normalizing annualized growth velocity in children with achondroplasia, we ultimately hope to improve the medical complications of disproportionate bone growth."

Another eminent person from Paediatrics, Ravi Savarirayan, added that "Children with achondroplasia are likely to experience a variety of medical complications caused by the condition. A treatment like vosoritide has the potential to decrease medical complications and to increase function and quality of life."