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BMN 333 — Competitive Intelligence Brief
phase 2
lysosomal enzyme replacement therapy
N-acetylgalactosaminidase (NAGLU)
Genetics and Rare Diseases
Small molecule
Live · refreshed every 30 min
Target snapshot
BMN 333 (BMN 333) — BioMarin Pharmaceutical. 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).
Comparator set
| Drug | Generic | Sponsor | Phase | Class | Target | Approval / PDUFA |
|---|---|---|---|---|---|---|
| BMN 333 TARGET | BMN 333 | BioMarin Pharmaceutical | phase 2 | lysosomal enzyme replacement therapy | N-acetylgalactosaminidase (NAGLU) |
Recent regulatory actions (last 90 days)
Upcoming PDUFA dates (next 180 days)
Patent timeline (forward window)
Sponsor landscape (lysosomal enzyme replacement therapy class)
- BioMarin Pharmaceutical · 1 drug in this class
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Cite this brief
Drug Landscape (2026). BMN 333 — Competitive Intelligence Brief. https://druglandscape.com/ci/bmn-333. Accessed 2026-05-13.
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