BioMarin Pharmaceutical (BMRN) investor relations material

Financial performance and growth

• Achieved 13% total revenue growth in 2025, with Voxzogo up 26% and enzyme therapies up 9%.

• Earnings per share grew faster than revenue, with over 2x leverage on the bottom line after adjustments.

• Expecting high single-digit revenue growth (7%-8%) in both enzyme therapies and skeletal conditions for 2026.

• Facing a 3% headwind from declining royalty and other revenue, but core business units remain strong.

• Quarterly revenue volatility is driven by timing of large international orders and inventory stocking, but patient additions remain steady.

Product pipeline and clinical milestones

• Phase III readouts for Voxzogo in hypochondroplasia and BMN 401 for ENPP1 deficiency expected in the first half of 2026.

• BMN 351 for Duchenne muscular dystrophy showed promising interim results, with final high-dose data expected later in 2026.

• BMN 333, a long-acting CNP, is being developed to potentially surpass Voxzogo and competitors in efficacy.

• CANOPY phase II trials are ongoing in ISS, Noonan’s, Turner’s, and SHOX, targeting a large but selective patient population.

Commercial strategy and competitive landscape

• Voxzogo 2026 revenue guidance is $975M-$1.025B, with key variables being international pricing and competitive switching.

• Only 25% of Voxzogo revenue is U.S.-based and subject to competition, representing about 7% of total revenue.

• Hypochondroplasia launch could begin in 2027, with a total addressable population of 14,000 due to lower diagnosis rates.

• Palynziq label expansion to adolescents opens a new segment, with 1,500 eligible U.S. patients and high adherence expected.

• No significant switching from Palynziq to new competitors observed; new entrants may expand the overall patient pool.