BioMarin Pharmaceuticals Inc BMRN

BioMarin Pharmaceutical Inc. es una empresa de biotecnología. La compañía desarrolla y comercializa productos farmacéuticos para diversas enfermedades y condiciones médicas. Hasta el 31 de diciembre de 2016, el portafolio terapéutico de la compañía consistía en cinco productos, y múltiples candidatos a productos clínicos y preclínicos. Sus productos comerciales incluyen Aldurazyme (laronidasa) para la Mucopolisacaridosis I (MPS I), Firdapse (amifampridina fosfato) para el Síndrome de Lambert Eaton Myasthenic (LEMS), Kuvan (sapropterina dihidrocloruro) para la fenilcetonuria (PKU), Naglazyme (galsulfasa) para la Mucopolisacaridosis VI (MPS VI) y Vimizim (elosulfasa alfa) para la Mucopolisacaridosis IV Tipo A (MPS IV A). La compañía está llevando a cabo ensayos clínicos sobre diversos candidatos a productos para el tratamiento de diversas enfermedades. Sus candidatos a productos clínicos incluyen Brineura, pegvaliasa, vosoritida, BMN 270 y BMN 250.

• GlobeNewswire Inc. 25 sept

  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.

  

• Zacks Investment Research 16 sept

  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.

  

• Zacks Investment Research 3 sept

  How BioMarin Turned a Competitive Threat Into a Royalty-Bearing Asset

  BioMarin settled a patent dispute with Ascendis Pharma over the achondroplasia drug Yuviwel, converting a competitive threat into a royalty stream. Under the agreement, BioMarin will receive 20% royalties on U.S. Yuviwel sales and 18% in the EU, Brazil, and South Korea through May 2030. While this monetizes competition, BioMarin still faces market pressure from Yuviwel and potential future competitors like BridgeBio's infigratinib.

  

• GlobeNewswire Inc. 25 ago

  Fabry Disease Treatment Market 2026-2031 - Growth Driven by Increasing Adoption of Enzyme Replacement Therapy

  The global Fabry disease treatment market is projected to grow from USD 2.63 billion in 2025 to USD 4.09 billion by 2031, with a CAGR of 7.63%. Growth is driven by expanded screening identifying more patients, advances in gene and mRNA therapies, and sustained enzyme replacement therapy demand. However, high treatment costs remain a significant barrier to patient access, particularly in emerging markets. Asia-Pacific is expected to record the fastest growth at 8.28% CAGR.