BioMarin Pharmaceutical (BMRN) investor relations material

Strategic priorities and commercial performance

• Focus on genetically defined rare diseases with nine commercial therapies across skeletal and metabolic conditions.

• Four products are tracking to blockbuster status, with VOXZOGO expected to reach $1–1.05 billion in 2024.

• Recent Amicus acquisition added GALAFOLD and PomOp, with peak sales potential of $1.4B and $1.2B in the mid-2030s.

• Substantial non-GAAP EPS accretion from new assets expected in 2027, supporting reinvestment in innovation.

Pipeline and R&D highlights

• VOXZOGO data in hypochondroplasia exceeded expectations and has been submitted to the FDA.

• Pipeline expanded with BMN 820 (FSGS, phase III, data in 2028) and ALE-01 (hypophosphatasia, substrate reduction therapy).

• BMN 333, a next-gen CNP analog for skeletal dysplasias, is in phase II with data expected next year.

• Internal innovation continues to drive pipeline growth.

VOXZOGO franchise and market dynamics

• Achondroplasia franchise shows strong growth, especially in the under-2 population, with 14% YoY revenue and 20% YoY patient demand.

• 90% patient retention on VOXZOGO despite YORVIPATH's entry; 10% switched in the U.S.

• Settlement with Ascendis secures royalties on YORVIPATH sales, clearing legal overhang.

• Full approval of VOXZOGO could enhance confidence and allow inclusion of extensive safety and efficacy data in the label.