Expert Interview
Digging into the recent Phase 3 results for Biomarin’s vosoritide (BMN 111) in achondroplasia
Ticker(s): BMRNA geneticist with experience in treating Children with Achondroplasia And Having Knowledge/Experience with Vosoritide.
Please describe your clinical practice, in particular with treating achondroplasia patients, and research in the space.
Added By: slingshot_insightsHow many patients do you treat? What treatment options do they have?
What is the mechanism of action of a C-type natriuretic peptide like vosoritide, how does it regulate bone growth?
Added By: slingshot_insightsWhat treatment options exist for patients whose growth plates are no longer open? According to the company, that’s 75% of the total patient population.
Added By: slingshot_insightsChange from baseline in growth velocity after one year of treatment with vosoritide, the primary endpoint, was 1.6 cm/yr. How satisfactory is this result?
Added By: slingshot_insightsNo clinically significant blood pressure decreases were reported. Was this something to be expected? Is it a strong point of Vosoritide vs competition?
Added By: slingshot_insightsHeight Z-score, body and limb proportionality and joint geometry; biochemical, biomarker and radiological assessments of bone growth and health; and evaluations of health-related quality of life (HRQoL), developmental status, and functional independence are still to be reported by the Company. What should we expect from those endpoints?
Added By: slingshot_insightsAre You Interested In These Questions?
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