Sean Lehmann

Sean Lehmann asked for BioMarin's latest thoughts on orphan drug exclusivity for VOXZOGO, particularly for kids under five, and the potential for switching to a competitor. He also inquired about BioMarin's balance sheet capacity and what it considers an efficient balance sheet structure, given its capital accumulation without business development.

Answer

Cristin Hubbard (Chief Commercial Officer, BioMarin Pharmaceutical) stated that for satisfied patients already on therapy, the majority are likely to remain on VOXZOGO, irrespective of orphan drug exclusivity, based on market research and adherence rates. Alexander Hardy (President and CEO, BioMarin Pharmaceutical) confirmed BioMarin submitted a petition to the FDA concerning orphan drug exclusivity for VOXZOGO. Brian Mueller (CFO, BioMarin Pharmaceutical) estimated BioMarin's total firepower for growth capital deployment at $4 billion-$5 billion, combining $2 billion cash on hand with leverageable earnings.

Sean Lehmann asked for BioMarin's latest thoughts on orphan drug exclusivity for Voxzogo, particularly for children under five, and the potential for patient switching to a competitor. He also inquired about BioMarin's balance sheet capacity and what the company considers an efficient balance sheet structure without business development.

Answer

Cristin Hubbard, Chief Commercial Officer, stated that market research suggests most satisfied patients are likely to remain on Voxzogo, irrespective of orphan drug exclusivity, due to its efficacy and safety. Alexander Hardy, President and CEO, confirmed BioMarin has submitted a petition to the FDA regarding Voxzogo's orphan drug exclusivity, with timing of the outcome tied to PDUFA. Brian Mueller, Chief Financial Officer, estimated BioMarin's total firepower for growth capital at $4 billion to $5 billion, leveraging $2 billion in cash and growing EBITDA, indicating a significant capacity for deployment.