New data on Vosorite for achondroplasia

BioMarin Research & Development day 2018, was presented 

on the 7th of November, 2018. Jonathan Day, BioMarin Executive Medical Director, presented "42-month data update with vosoritide for achondroplasia", and stated that phase 3 was completed on the 7th November and with a full enrolment of participants.

First, it was shown how Vosoritide development is covered, presenting the core of the research:

Image 1. Four Pillars Underpin Vosoritide Program. Credits: BioMarin 

It was then presented the full program, with the summary of all the studies conducted and many still ongoing

Image 2. Comprehensive Vosoritide Achondroplasia Clinical Program. Credits: BioMarin

For phase 2, studies 111-202, BioMarin presented data of the group of patients (cohort 3), having a daily dose of 15 µg/kg of Vosoritide. This data showed an increase in average growth velocity during 42 months, measured in 6 months periods. The average increase in every 6 months varied, as seen in the image below, between 1 to 2 cm more over the natural growth, or baseline (BL). This sums up in an average of 5,7 cm growth velocity increase over a period of 42 months (3,5 years).

Image 3. Durable Growth Sustained through 42 months Cohort 3 at 15 µg/kg. Credits: BioMarin

Regarding phase 3, study 111-301, which includes 110 children from 5 to 18 years old, it aims to collect the change of Average Growth Velocity from baseline at 52 weeks (1 year). The company expects to present these results at the end of 2019. There was also the presentation of data related to the biomarker, collagen x, during a 24 months period. A biomarker is a measurable indicator of a specific biological state or condition and the quantity of collagen X protein detected in the body is related to the increase of hypertrophic chondrocytes, that exist in the growth plate of the long bones. So, an increase in collagen X correlates with an increase in the chondrocytes of the growth plate, that are needed for new bone formation.

Overall, Vosoritide was generally well-tolerated across all doses, with blood pressure and heart rate reductions reported as transient (i.e. resolved within minutes), and without medical intervention.

Looking forward to next developments of phase 3.