8-K filing — BIOMARIN PHARMACEUTICAL INC (BMRN)

Enzyme Replacement Therapy Market to Grow to USD 27.90 Billion by 2035 at a CAGR of 8.59% - SNS Insider

The global Enzyme Replacement Therapy (ERT) market is projected to grow from USD 12.24 billion in 2025 to USD 27.90 billion by 2035, with a CAGR of 8.59%. Growth is driven by early identification of rare lysosomal storage disorders, increased newborn screening, orphan drug incentives, and expanding healthcare access. North America leads with 38.76% market share, while Asia-Pacific shows the fastest growth at 9.43% CAGR. Imiglucerase dominates products, and Gaucher disease represents the largest therapeutic condition.

BioMarin Stops Voxzogo Development for Noonan Syndrome

BioMarin Pharmaceutical announced it is discontinuing development of Voxzogo for Noonan syndrome due to study feasibility and treatment landscape concerns, though not due to safety or efficacy issues. Despite this setback, Voxzogo generated $472 million in first-half 2026 sales (up 9% YoY) and is on track to achieve blockbuster status with expected 2026 sales of $1.00-$1.05 billion. The drug faces increasing competition from Ascendis Pharma's Yuviwel and BridgeBio's infigratinib, though BioMarin secured a royalty-bearing settlement with Ascendis.

BioMarin Pharmaceutical shares are trading lower after the company announced its decision to discontinue development of VOXZOGO for children with Noonan syndrome.

BioMarin Pharmaceutical Decides To Discontinue Development Of VOXZOGO For Children With Noonan Syndrome

On September 15, 2026, BioMarin Pharmaceutical Inc. (BioMarin) announced its decision to discontinue development of VOXZOGO for children with Noonan syndrome. BioMarin made this decision after assessing a number of