BioMarin Pharmaceutical

BioMarin stands tall with first approved drug for rare disease that causes dwarfism

The European Commission has approved a BioMarin Pharmaceutical drug that treats achondroplasia, a rare genetic disorder that slows bone growth and is the most common cause of dwarfism. The commission also granted conditional approval to a cancer drug from partners Incyte and MorphoSys.

BioMarin Pharmaceutical Signs an Agreement with Deep Genomics on Advancing Programs Identified Using Artificial Intelligence

Shots: Deep Genomics will receive an undisclosed upfront payment and is eligible to receive development milestones, BioMarin will receive an exclusive option to obtain Deep Genomics’ rights to each program for development & commercialization Deep Genomics will use its AI drug discovery platform (The AI workbench) to identify & validate target mechanisms, lead candidates & …

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FDA rejects BioMarin’s hemophilia A gene therapy in ‘massive surprise’

In a CRL issued for valoctocogene roxaparvovec, the FDA told BioMarin that differences between its Phase I/II and Phase III studies limited the ability to rely on the earlier trial to support durability of effect, which an analyst said likely referred to a product comparability issue.