Monday, August 3, 2015

Biomarin announces the first patient in the fourth cohort of the phase 2 study

During the second quart financial results conference today, Biomarin announced the first patient included in the fourth cohort of the phase 2 study, at a 30mcg/kg/day dosing.
Furthermore, Biomarin announced that is planning to conduct the pivotal study (for registry) at the dose of 15mcg/kg/day, which has leaded to a 50% increase in growth velocity. Once approved by the regulatory authorities, they plan to start the study (phase 3) in the first semester of 2016.

This is the excerptum from the today's press release:
"Another highly anticipated event in the second quarter was the completion and results of the Phase 2 study with vosoritide for the treatment of achondroplasia. We were pleased to share that vosoritide increased the mean annualized growth velocity by 50% in children in the highest dose cohort who received 15 micrograms per kilogram daily. We believe growth velocity is a leading indicator of improvement in many of the severe complications associated with the disorder, such as foramen magnum compression, sleep apnea, bowed legs, spinal stenosis, recurrent ear infections and obesity.  Based on efficacy and safety observed in the first 3 cohorts of the Phase 2 study we are planning for registration enabling studies at 15 micrograms per kilogram daily and have also begun enrollment in a 4th higher dose cohort with 30 micrograms per kilogram daily." 
This is the link for the complete press release. 

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