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BMN 333
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).
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). Used for Mucopolysaccharidosis type IIIB (MPS IIIB).
At a glance
| Generic name | BMN 333 |
|---|---|
| Sponsor | BioMarin Pharmaceutical |
| Drug class | lysosomal enzyme replacement therapy |
| Target | N-acetylgalactosaminidase (NAGLU) |
| Modality | Small molecule |
| Therapeutic area | Genetics and Rare Diseases |
| Phase | Phase 2 |
Mechanism of action
By inhibiting NAGLU, BMN 333 aims to reduce the accumulation of glycosaminoglycans in cells, thereby alleviating the symptoms of MPS IIIB. This approach is based on the understanding that the deficiency of NAGLU leads to the accumulation of these molecules, causing cellular damage and disease progression.
Approved indications
- Mucopolysaccharidosis type IIIB (MPS IIIB)
Common side effects
- Injection site reactions
- Abdominal pain
- Nausea
Key clinical trials
- A Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia (PHASE2, PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- BMN 333 CI brief — competitive landscape report
- BMN 333 updates RSS · CI watch RSS
- BioMarin Pharmaceutical portfolio CI