BioMarin Pharmaceutical touts $3.2B 2025 revenue, Voxzogo surge and Amicus deal at JPM Conference

BioMarin Pharmaceutical (NASDAQ:BMRN) used its presentation at the 44th Annual J.P. Morgan Healthcare Conference to outline strategic priorities for 2026, provide preliminary 2025 revenue figures, and highlight a slate of upcoming regulatory and clinical catalysts. Chief Executive Officer Alexander Hardy said the company has been “transforming BioMarin” over the past 24 months and is targeting an additional period of growth driven by Voxzogo, its enzyme therapies portfolio, and the pending acquisition of Amicus.

Strategy refresh and 2026 priorities

Hardy framed BioMarin’s strategy around three pillars introduced at the company’s September 2024 Investor Day: innovation, growth, and a “value commitment” focused on operating leverage. He said BioMarin has prioritized pipeline programs it views as having the most transformative potential and pointed to accelerated progress for two pipeline assets, BMN 333 and BMN 351.

For 2026, Hardy said BioMarin’s priorities are to:

• Accelerate revenue by expanding Voxzogo geographically and through future indications, driving growth in enzyme therapies, and pursuing a potential label expansion for Palynziq in adolescents.
• Advance innovation with multiple data readouts and regulatory events, including a newly announced filing for full approval of Voxzogo in achondroplasia.
• Strengthen the pipeline through business development, with an emphasis on earlier-stage deals as the company focuses on deleveraging following the Amicus financing.

Preliminary 2025 revenue and Voxzogo performance

Hardy announced BioMarin’s estimated preliminary 2025 revenue of $3.2 billion. He also provided a preliminary 2025 revenue figure for Voxzogo of $920 million, describing the product’s performance as strong and indicating that, on a quarter-by-quarter basis, the implied fourth-quarter year-over-year growth rate would be 27%.

In the Q&A portion of the session, Hardy said Voxzogo’s growth in achondroplasia is being supported by continued launches in additional countries and deeper penetration in existing markets. He emphasized the importance of the drug’s indication in infants aged zero to two years, noting that treatment decisions are increasingly made early, sometimes before birth, and initiated within weeks or months of delivery.

Hardy said BioMarin has previously discussed adherence rates around 90%, describing them as “extremely high” across markets and attributing that to the drug’s importance and the ability of families to incorporate daily injections into routines.

Amicus acquisition: growth and portfolio fit

Hardy briefly discussed BioMarin’s pending acquisition of Amicus, announced Dec. 19, with an equity value of $4.8 billion. He characterized the deal as a strategic fit that “drops perfectly” into BioMarin’s enzyme therapies business and said it should accelerate and diversify revenue growth, creating four major growth drivers in the combined portfolio.

Hardy said BioMarin expects the transaction to begin to be accretive within 12 months post-close and “substantially accretive” beginning in 2027. He also said the company expects the deal to close in the second quarter.

Hardy highlighted two key products:

• Galafold (Fabry disease): Described as the only oral therapy in Fabry disease. Hardy said it is currently available in about 40 countries and BioMarin intends to expand access within its 80-country footprint. He cited Fabry prevalence of about 100,000 patients globally, with about 18,000 diagnosed and 12,000 treated, suggesting opportunity to increase diagnosis and treatment rates.
• Pombiliti and Opfolda (Pompe disease): Hardy said the products are reimbursed in 15 countries and that BioMarin sees growth potential through broader geographic reach and “real-world data” supporting switching where on-label. He also cited potential label expansions, including expansion within late-onset Pompe disease and a possible move into infantile-onset Pompe disease.

Hardy said BioMarin is not providing updated projections for the enzyme therapies growth rate during the presentation, but indicated confidence it could outperform prior “high single-digit” growth commentary after the Amicus transaction closes and is integrated.

Clinical and regulatory catalysts: Voxzogo, Palynziq, BMN 351, and BMN 333

Head of R&D Greg Friberg outlined what he called a “catalyst-rich” year, including three major data readouts, label and age expansions, and new data releases for BMN 351 and BMN 333.

Voxzogo full approval filing: Friberg said BioMarin plans to file with the FDA for full approval of Voxzogo in the first half of the year after completing post-marketing requirements. He said the submission will draw on more than 10,000 patient-years of safety data and include efficacy endpoints beyond height, such as body morphometry and anatomical changes. He also noted BioMarin is “very excited” about foramen magnum data expected to be shared later in the year.

Palynziq adolescent expansion: Friberg noted an FDA PDUFA action date of Feb. 28 for an adolescent label expansion. He said results were effective and similar to adults in lowering phenylalanine levels and enabling increased intact food consumption.

BMN 351 (Duchenne muscular dystrophy): Friberg said the company has “turned over the cards” for the first two cohorts and observed encouraging, dose-dependent dystrophin responses across multiple assays (including Western blot and mass spectrometry). The 12 mg/kg cohort has been initiated, with additional data expected before year-end, including longer-term safety and an initial look at functional measures such as stride velocity 95C. Friberg said results for the 6 and 9 mg/kg cohorts are expected in an oral presentation at the Muscular Dystrophy Association meeting in March.

BMN 333 (long-acting CNP for achondroplasia): Friberg presented newly released pharmacokinetic data showing increases in free CNP exposure that exceeded the company’s target of a threefold AUC improvement, including a highest cohort showing more than a 13-fold increase. He said BioMarin plans to initiate a combined phase 2/3 study in the first half of the year, benchmarking three doses against Voxzogo before advancing into a phase 3 head-to-head study seeking superiority on annualized growth velocity, while also measuring proportionality, anatomical changes, and mobility.

Competitive dynamics and IP strategy for Voxzogo

In Q&A, Hardy said BioMarin plans to “vigorously defend” its intellectual property around CNP in achondroplasia. He said BioMarin has petitioned the FDA regarding orphan drug exclusivity, requesting that approval of a product “currently under review” be delayed until the company’s orphan exclusivity expires, which he said has three years remaining. He also pointed to ongoing IP litigation, including an expected decision from the U.S. International Trade Commission and additional litigation activity in the U.S. and potentially Europe.

Hardy said that when additional treatment options enter the market, switching decisions will likely be driven by a shared discussion between healthcare providers and caregivers, with safety and efficacy as primary factors and convenience as a secondary consideration. He also pointed to BMN 333 as a potential longer-term “game changer” for achondroplasia based on the exposure profile shown.

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.