BioMarin Pharmaceutical (BMRN)

Business Overview

BioMarin is a profitable rare-disease company whose fastest-growing drug matters without dominating. FY2025 revenue was $3,221M and GAAP net income $349M. Charges for withdrawing ROCTAVIAN, its gene therapy for haemophilia A, produced a Q4 GAAP net loss of $(46.6)M. VOXZOGO, for the dwarfism condition achondroplasia, brought in $926.9M (+26% YoY), 28.8% of revenue. Cash and investments were ~$2.05B at year-end 2025.

On 27 April 2026, after these figures, BioMarin completed its acquisition of Amicus Therapeutics, whose GALAFOLD treats Fabry disease and POMBILITI + OPFOLDA treats Pompe disease, so later periods sit on a larger revenue base.

At 28.8%, VOXZOGO is meaningful concentration but far below Vertex's cystic fibrosis drugs (92.9% of its revenue) or Moderna's COVID vaccines (93.1%). Approval for new uses of VOXZOGO and follow-on programmes are the main pipeline levers.

How the Numbers Read

R&D was $922M in FY2025. BMN 333 is a long-acting form of C-type natriuretic peptide (CNP), the growth pathway VOXZOGO also targets. At the FY2025 results it was due to start Phase 2/3 in the first half of 2026, after Phase 1 drug levels exceeded the target. By the Q2 2026 results (August 2026) the study was enrolling, with a data update expected in 2027.

The ROCTAVIAN withdrawal explains the Q4 loss. Treat those charges as one-off: annualising the Q4 loss would badly understate the earnings of a company that was profitable for the year.

BioMarin does not burn cash like Moderna (~$468M a quarter), but the runway guide still works as a liquidity check. About $2.05B of cash and investments funds Phase 2/3 spend without the acute financing risk of a pre-revenue company with <4 quarters of cash.

Valuation Framework

Value the parts separately: the commercial rare-disease portfolio on filed FY2025 revenue ($3,221M), with VOXZOGO ($926.9M) as the growth driver, plus a risk-adjusted NPV (rNPV) on BMN 333 and other disclosed programmes at a 10.0% discount rate, with the probability of approval in the cash flows.

Peak-sales figures for VOXZOGO are analyst estimates, not company guidance: ~$1.5B from Evercore ISI (via trade press), and more than $2B with new approved uses in other trade-press reports. Start from the $926.9M run-rate and layer peak scenarios on top in the model, never in the filed figures.

What to Watch in the Financials

VOXZOGO's share (28.8%). $926.9M of $3,221M revenue, growing 26%. A slowdown would show quickly in group growth, because pipeline programmes add revenue only after approval.

BMN 333 Phase 2/3 (enrolling; data update expected 2027). The main pipeline milestone. Model it with explicit phase probabilities.

Key Risks

BMN 333 clinical attrition. Rare-disease programmes have their own success rates: 17.0% from Phase I to approval in a BIO study of 2011-2020, against 7.9% for all indications. Use the rare-disease rate where it is sourced.

The ROCTAVIAN precedent. A programme failure or label reversal can bring one-off charges again, even in a profitable year.