BioMarin Pharmaceutical (NASDAQ:BMRN) executives used a fireside chat at TD Cowen’s 46th Annual Healthcare Conference to outline the company’s refreshed strategy, discuss upcoming competitive dynamics for Voxzogo, and frame key growth and margin expectations tied to its core rare disease franchises and recent acquisitions.
Strategy refresh emphasizes business development and scaled rare-disease infrastructure
Chief Executive Officer Alexander Hardy said that since becoming CEO two years ago, he led a “strategic refresh” that elevated business development (BD) as a more significant contributor to BioMarin’s future. He pointed to two transactions completed last year—Inozyme and the pending Amicus acquisition—as evidence of that shift. Hardy said both acquisitions are expected to fit into BioMarin’s existing enzyme therapy business, which he described as operating at scale across 80 countries.
Looking ahead, Hardy said BioMarin expects to continue pursuing BD, primarily aimed at strengthening the pipeline. He added that the company will need to delever following the Amicus acquisition before pursuing anything “very significant,” but expects to continue doing earlier-stage pipeline deals in the meantime.
2026 framework includes headwinds from other revenue, ROCTAVIAN removal, and Kuvan erosion
Chief Financial Officer Brian Mueller expanded on management’s view of 2026, noting that the company’s 2026 revenue framework includes a roughly 3% headwind tied to declines in “other revenue.” He attributed the headwind to several factors, including the removal of ROCTAVIAN revenue expectations, continued erosion of Kuvan revenue (generic in the U.S. for more than five years), and lower royalty revenue from an older out-license partnership that has now run through its royalty period.
Mueller said that when adjusting for that headwind, BioMarin expects both core business units to grow at high single-digit rates at the midpoint, citing:
- Enzyme therapies: 7% growth at the midpoint
- Skeletal conditions: 8% growth at the midpoint (including competition)
Key risks: order timing, macro uncertainty, and Voxzogo switching behavior
Asked what could cause results to fall short versus guidance, Hardy highlighted execution and timing dynamics in the enzyme therapy business, which he described as a more than $2 billion franchise. He said ordering can vary meaningfully because many customers are national healthcare systems, creating quarter-to-quarter timing variability. He also cited monitoring geopolitical and global economic risks.
For skeletal conditions, Hardy said the major variable is the “ultimate impact” of competition on Voxzogo, noting a competitor was approved by the FDA on the prior Friday. He said BioMarin’s guidance assumes competition across all points, with greater or earlier switching pressure reflected toward the lower end of the range.
On potential upside, Hardy said outperformance could come from continued growth in the under-two population—where BioMarin is currently the only approved therapy—along with international expansion. He said only about 25% of Voxzogo revenue is generated in the U.S., and that BioMarin is still deepening penetration in existing markets and launching into new countries.
Margin outlook: 40% operating margin target in 2026 excluding Amicus impact
Mueller said BioMarin believes a 40% operating margin in 2026 is achievable, excluding the impact of Amicus (which he said would be slightly dilutive). He attributed margin expansion to a financial strategy refined during the 2024 strategic review, including completion of a $0.5 billion cost transformation program. Mueller said the company has constrained operating expense growth while continuing to invest, and described recent performance as showing “leveraged growth,” with profitability rising at roughly two to three times the rate of revenue growth.
On Amicus, Mueller said BioMarin expects to close the acquisition in Q2 and that the exact timing within the quarter could be a swing factor. He said it was “not unreasonable” to model $3.7 billion of total revenue and $4.80 of non-GAAP EPS for 2026 with Amicus included for the second half of the year, while emphasizing that BioMarin would provide an updated view later and that earlier comments excluded Amicus.
Mueller also noted BioMarin disclosed $0.25 of loss per share related to pre-closing Amicus costs, including interest expense from raised debt and transaction fees. Hardy said BioMarin believes Amicus assets can reach more patients in more geographies within BioMarin’s commercial infrastructure, and reiterated the company’s intent to provide more detail on the deal’s benefits post-close.
Pipeline and franchise updates: Voxzogo competition, hypochondroplasia readout, BMN 333, and BMN 401
Hardy said BioMarin has prepared for the Voxzogo competitive period over the last two years and believes switching will be “complicated” and play out over time. He cited the importance to caregivers and physicians of long-term durability and safety, and said BioMarin has extensive evidence on Voxzogo. He also said 90% of Voxzogo patients are adherent and that injections have become part of daily family routines, making convenience less central to decision-making than durability and safety.
Hardy said BioMarin has a “five-year head start” and described a U.S. clinical coordinator team that trains families and maintains ongoing contact. He also said BioMarin has “eyes on every single patient in the United States with achondroplasia.”
Regarding the ITC matter, Hardy said the case is expected to be heard in April, with a preliminary decision anticipated around August.
Hardy also previewed several clinical catalysts:
- Hypochondroplasia Phase 3 (Voxzogo): BioMarin powered the study for 1.5 cm annualized growth velocity (AGV), referencing proof-of-concept data showing 1.8 cm. Hardy said there are scientific reasons to believe AGV could be better than in achondroplasia and estimated a total addressable global population of about 14,000 diagnosed patients (with genetic prevalence cited as ~24,000).
- BMN 333 (achondroplasia): Hardy said BioMarin aims to deliver weekly dosing and superior efficacy. He said a healthy volunteer PK study exceeded an internal threshold of 2x–3x free CNP levels versus other long-acting CNP programs, with the highest dose cohort reaching 13x. BioMarin plans an operationally seamless Phase 2/3 design, selecting from three dose cohorts for Phase 2 and then taking a single dose into Phase 3 head-to-head versus Voxzogo. He said the study is powered for 2.25 cm AGV, described as ~50% better than Voxzogo.
- BMN 401 (ENPP1), from Inozyme: Hardy said a pediatric Phase 3 readout is expected in the first half of the year, with co-primary endpoints of phosphate levels and a radiographic functional endpoint. He estimated a conservative 2,000–2,500 patient opportunity, with about 70% of patients in the adult population, and said an adult Phase 3 would be designed based on pediatric results, with a potential adult approval timeline in the early 2030s.
Finally, Hardy addressed Palynziq, noting its label was expanded to adolescents over the weekend. He said Palynziq grew 22% last year and argued the adolescent indication could drive meaningfully higher penetration than the approximately 10% increase in addressable population might suggest, because adolescents are more likely to be under specialist care and motivated to control phenylalanine levels as they approach adulthood and independence.
About BioMarin Pharmaceutical (NASDAQ:BMRN)
BioMarin Pharmaceutical Inc is a biopharmaceutical company specializing in the development and commercialization of therapies for rare genetic and metabolic diseases. The company focuses on addressing unmet medical needs by leveraging enzyme replacement therapy, small molecule pharmacological chaperones and gene therapy technologies. Headquartered in Novato, California, BioMarin operates research and development facilities in the United States and Europe.
The company’s commercial portfolio includes several approved therapies targeting inherited disorders.
