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BioMarin Pharmaceutical — Portfolio Competitive Intelligence Brief
1 marketed
0 filed
8 Phase 3
6 Phase 2
6 Phase 1
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Portfolio leaders (marketed + Phase 3 / filed)
| Drug | Generic | Phase | Class | Target | Area | First approval |
|---|---|---|---|---|---|---|
| RDD to Palynziq | RDD to Palynziq | marketed | Enzyme replacement therapy | Phenylalanine ammonia-lyase (PAL) | Metabolic/Genetic disorder | |
| BMN 110 | BMN 110 | phase 3 | Gene therapy (AAV-based) | HEXA gene | Neurology / Rare Genetic Disorder | |
| BMN 110 - Every Other Week | BMN 110 - Every Other Week | phase 3 | Enzyme replacement therapy | Lysosomal acid lipase (LAL) | Rare genetic metabolic disorder | |
| BMN 165 | BMN 165 | phase 3 | Gene therapy (AAV-based) | GALNS gene (N-acetylgalactosamine-6-sulfatase) | Rare Genetic Disorder / Lysosomal Storage Disease | |
| BMN 110 - Weekly | BMN 110 - Weekly | phase 3 | Enzyme replacement therapy | Lysosomal acid lipase (LAL) | Rare genetic metabolic disorder | |
| N-acetylgalactosamine 4-sulfatase | N-acetylgalactosamine 4-sulfatase | phase 3 | Enzyme replacement therapy | GALNS (N-acetylgalactosamine 4-sulfatase) | Rare genetic disease / Lysosomal storage disorder | |
| Placebo/rhASB | Placebo/rhASB | phase 3 | Enzyme replacement therapy | Arylsulfatase B (ARSB) | Rare genetic disease / Lysosomal storage disorder | |
| sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin | sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin | phase 3 | ||||
| BMN 111 | BMN 111 | phase 3 | CNP analog | Natriuretic peptide receptor B (NPR-B) | Rare genetic disorders / Endocrinology |
Therapeutic area mix
- Rare genetic disease / Lysosomal storage disorder · 2
- Rare genetic metabolic disorder · 2
- Neurology / Rare Genetic Disorder · 1
- Other · 1
- Genetics and Rare Diseases · 1
- Rare genetic disorders / Endocrinology · 1
- Rare Genetic Disorder / Lysosomal Storage Disease · 1
- Metabolic/Genetic disorder · 1
Recent regulatory actions (last 90 days)
Upcoming PDUFA dates (next 180 days)
Patent timeline (forward window)
Competitive overlap (companies sharing drug classes)
- Alexion Pharmaceuticals, Inc. · 1 shared drug class
- Cambium Bio Limited · 1 shared drug class
- Eurofarma Laboratorios S.A. · 1 shared drug class
- Genzyme, a Sanofi Company · 1 shared drug class
- ISU Abxis Co., Ltd. · 1 shared drug class
- JCR Pharmaceuticals Co., Ltd. · 1 shared drug class
- Masonic Cancer Center, University of Minnesota · 1 shared drug class
- Pfizer Inc. · 1 shared drug class
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Cite this brief
Drug Landscape (2026). BioMarin Pharmaceutical — Portfolio Competitive Intelligence Brief. https://druglandscape.com/ci/company/biomarin-pharmaceutical. Accessed 2026-06-10.