BioMarin Pharmaceutical

Quick facts

Marketed products

• RDD to Palynziq · Metabolic/Genetic disorder
  Palynziq is a recombinant phenylalanine ammonia-lyase (PAL) enzyme that breaks down phenylalanine, reducing blood phenylalanine levels in patients with phenylketonuria.

Phase 3 pipeline

• BMN 110 · Neurology / Rare Genetic Disorder
  BMN 110 is a gene therapy that delivers a functional copy of the HEXA gene to restore hexosaminidase A enzyme activity in patients with Tay-Sachs disease.
• BMN 110 - Every Other Week · Rare genetic metabolic disorder
  BMN 110 is a recombinant human lysosomal acid lipase (LAL) enzyme replacement therapy that restores deficient enzyme activity in patients with LAL deficiency.
• BMN 110 - Weekly · Rare genetic metabolic disorder
  BMN 110 is a recombinant human lysosomal acid lipase (LAL) enzyme replacement therapy that restores deficient enzyme activity in patients with LAL deficiency.
• BMN 111 · Rare genetic disorders / Endocrinology
  BMN 111 is a C-type natriuretic peptide (CNP) analog that activates natriuretic peptide receptor B (NPR-B) to promote bone growth and increase height.
• BMN 165 · Rare Genetic Disorder / Lysosomal Storage Disease
  BMN 165 is a gene therapy that delivers a functional copy of the GALNS gene to restore enzyme activity deficient in mucopolysaccharidosis IVA (Morquio A syndrome).
• N-acetylgalactosamine 4-sulfatase · Rare genetic disease / Lysosomal storage disorder
  N-acetylgalactosamine 4-sulfatase is an enzyme replacement therapy that replaces the deficient GALNS enzyme to break down keratan sulfate and chondroitin 6-sulfate in lysosomal storage disease.
• Placebo/rhASB · Rare genetic disease / Lysosomal storage disorder
  rhASB (recombinant human arylsulfatase B) is an enzyme replacement therapy that replaces deficient arylsulfatase B to break down glycosaminoglycans accumulated in Maroteaux-Lamy syndrome.
• sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin

Phase 2 pipeline

• BMN 333 · Genetics and Rare Diseases
  BMN 333 is a small molecule that targets the enzyme alpha-N-acetylgalactosaminidase (NAGLU), which is deficient in patients with mucopolysaccharidosis type IIIB (MPS IIIB).
• Phenoptin · Other
• rAvPAL-PEG
• Sapropterin Dihydrochloride and Vitamin C
• Vosoritide Injection
• Vosoritide Injection [Voxzogo]

Phase 1 pipeline

• BMN 190
• BMN 307
• BMN 349
• BMN 351
• PRO044 IV
• PRO044 SC

Competitive intelligence

For the full portfolio CI brief — phase mix, recent regulatory actions, upcoming PDUFA dates, patent timeline, class-overlap with competitors:

• BioMarin Pharmaceutical portfolio CI brief
• BioMarin Pharmaceutical pipeline updates RSS