By Fineline Cube 
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced during its third‑quarter earnings call that it no longer expects to hit its USD 4 billion revenue target by 2027. The company cited slowing uptake of its gene‑therapy product Roctavian and the looming entry of competitors for its achondroplasia therapy Voxzogo as the primary drivers of the revised outlook.
Roctavian Divestment and Sales Slump
- Product Background – Roctavian, an AAV5‑vector‑based gene therapy approved by the FDA in June 2023, delivers a functional copy of the Factor VIII gene to achieve a one‑time cure for hemophilia A.
- Market Performance – The therapy generated USD 26 million in revenue in 2024, far below expectations. In August 2024, BioMarin scaled back commercialization to the U.S., Germany, and Italy, laid off ~225 employees, and shut its gene‑therapy manufacturing site.
- Cost Reduction Plan – Annual direct spending on Roctavian is being trimmed to ~USD 60 million, and new patient enrollment for clinical trials has been halted. The company is now preparing to divest the product line.
Voxzogo Faces Growing Competition
- Current Position – Voxzogo, a subcutaneous CNP analog for achondroplasia, grew 15 % YoY in Q3, adding USD 218 million—nearly one‑third of BioMarin’s USD 776 million total revenue.
- Competitive Landscape – Two challengers, Ascendis Pharma and BridgeBio, are rapidly developing alternative therapies, threatening Voxzogo’s monopoly on the most common form of dwarfism.
- Revenue Forecast – Executives “informally” indicated that 2027 revenue could fall between USD 3.65 billion and USD 4 billion, reflecting the potential erosion of Voxzogo’s market share.
Strategic Outlook
- BioMarin is reassessing its portfolio, prioritizing high‑margin projects and exploring divestiture of low‑performing assets.
- The company remains focused on accelerating other gene‑therapy candidates and expanding access to existing products in under‑served markets.
Forward‑Looking Statements
This release contains forward‑looking statements that involve risks and uncertainties. Actual results may differ materially.-Fineline Info & Tech