Phase III study of BMN 111 shows efficacy maintained in 2 years of treatment for achondroplasia.- BioMarin Pharma

BioMarin Pharmaceutical announced that data from the open-label long-term extension of the Phase III study of 15 µg/kg dose of BMN 111 (vosoritide) for the treatment of achondroplasia showed that children maintained an increase in Annual Growth Velocity (AGV) through the second year of continuous treatment with vosoritide. Children who received two years of vosoritide therapy had a baseline mean AGV of 4.28 cm/year. After one year of treatment, mean AGV was 5.71 cm/year and after the second year mean AGV was 5.65 cm/year, demonstrating sustained restoration of a major portion of the growth deficit in achondroplasia through the second year of treatment. Children also had an improved height z-score, which is a measure of height relative to that of a similar population of average height. In the children who were crossed over from placebo to vosoritide in the open-label extension arm, similar efficacy after one year was observed compared to children who received treatment with vosoritide after one year. Children who were crossed over to treatment had a baseline mean AGV of 3.99 cm/year. After one year of treatment, mean AGV was 5.57 cm per year. Retention of subjects on treatment was high with 93% of patients originally randomized to receive vosoritide remaining on treatment two years later. Data were was presented at an oral presentation at ENDO21, the Endocrine Society's Annual Meeting by Professor Ravi Savarirayan, M.B., B.S., M.D., clinical investigator from the Murdoch Children's Research Institute, Royal Children's Hospital, University of Melbourne, Parkville, Victoria.