BioMarin impresses with Phase II dwarfism drug
This article was originally published in Scrip
BioMarin Pharmaceuticals has raised expectations for its dwarfism product BMN-111 (vosoritide) on the back of strong Phase II data.
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R&D president Hank Fuchs said vosoritide can be filed based on height gains for children and safety in babies and toddlers with the most common form of dwarfism, but timing depends on meetings with regulators.
Pfizer adds to its rare disease portfolio with purchase of Therachon, a Swiss company working in achondroplasia. But whether it can catch up with Biomarin’s Phase III vosoritide is still to be seen.
Emerging Company Profile: Switzerland-based Therachon is developing therapeutics for rare genetic pediatric diseases with a lead program in achondroplasia, also known as genetic dwarfism. CEO Luca Santarelli explains the mechanism behind lead molecule TA-46 and plans moving forward.