BioMarin Outlines Strategy Refresh, Voxzogo Competition, and 40% Margin Goal

Executives detail pipeline updates, financial targets, and plans for acquisitions at healthcare conference

Published on Mar. 7, 2026

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BioMarin Pharmaceutical executives used a recent healthcare conference to outline the company's refreshed strategy, discuss upcoming competitive dynamics for its drug Voxzogo, and frame key growth and margin expectations tied to its rare disease franchises and recent acquisitions. The leadership team highlighted plans to continue pursuing business development deals, projected high single-digit growth for its core business units, and set a 40% operating margin target for 2026 excluding the impact of the pending Amicus acquisition.

Why it matters

BioMarin's strategy refresh and financial targets demonstrate its efforts to diversify its pipeline and maintain profitability in the face of headwinds like generic competition and the removal of certain revenue streams. The company's plans to strengthen its rare disease infrastructure through acquisitions and pipeline advancements could position it for continued growth, though it will need to navigate competitive pressures for key products like Voxzogo.

The details

CEO Alexander Hardy said the company's strategic refresh has elevated business development as a more significant contributor to BioMarin's future, pointing to recent acquisitions of Inozyme and Amicus. The company now sees 'multiple pillars of growth,' including its enzyme therapies and a skeletal conditions business anchored by Voxzogo. CFO Brian Mueller projected a 3% revenue headwind in 2026 from declines in 'other revenue,' but said the core business units are expected to grow at high single-digit rates. On margins, Mueller said BioMarin believes a 40% operating margin in 2026 is achievable, excluding the Amicus impact, citing a $0.5 billion cost transformation program. Regarding Voxzogo, Hardy said BioMarin has prepared for competition and believes switching will be 'complicated' due to the importance of long-term durability and safety. The company also previewed several pipeline updates, including a Phase 3 readout for its hypochondroplasia drug and plans for a head-to-head study of BMN 333 against Voxzogo.

  • The ITC matter regarding Voxzogo is expected to be heard in April, with a preliminary decision anticipated around August.
  • A pediatric Phase 3 readout for BMN 401 (ENPP1) is expected in the first half of 2026.

The players

BioMarin Pharmaceutical

A biopharmaceutical company specializing in the development and commercialization of therapies for rare genetic and metabolic diseases.

Alexander Hardy

Chief Executive Officer of BioMarin Pharmaceutical.

Brian Mueller

Chief Financial Officer of BioMarin Pharmaceutical.

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