BioMarin Pharmaceutical has reported positive results from a Phase II proof-of-concept and dose finding study of BMN 111 (vosoritide) in children with achondroplasia, a common form of human dwarfism.
Vosoritide is an analogue of C-type Natriuretic Peptide (CNP) that is being studied in children with achondroplasia under the age of 18 years as their bones are still amenable to growth.
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In the trial, data from the 26 children showed a favourable safety profile and efficacy at the 15µg/kg/daily dose.
The ten children in cohort three treated with 15µg/kg a day had a mean increase of 50% in their annualised growth velocity compared against annualised prior six month natural history baseline growth velocity, according to the company.
BioMarin clinical development vice-president Dr Wolfgang Dummer said: "In children receiving the highest dose of 15µg/kg daily, we observed a 50% increase in mean annualised growth velocity compared to their own natural history control growth velocity.
The Phase II trial was an open-label and sequential cohort dose-escalation study of BMN 111 in children with achondroplasia, with an average age of 7.8 years.
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By GlobalDataPatients have been treated with either 2.5µg/kg/daily, 7.5µg/kg/ daily or 15µg/kg/ daily, respectively, in this three dose cohort study.
All subjects participating in the study were now been switched to the highest dose of 15µg/kg/ daily for the duration of the 18 month extension study, based on the safety profile observed to date across the three dose cohorts.
BioMarin chairman and CEO Jean-Jacques said: "It is estimated that about 96,000 patients in our established territories are afflicted with achondroplasia, so approximately 25%, or 24,000, are under 18 years of age and in our addressable market."
