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BMN 110 - Every Other Week
BMN 110 - Every Other Week is a Enzyme replacement therapy Small molecule drug developed by BioMarin Pharmaceutical. It is currently in Phase 3 development for Lysosomal acid lipase (LAL) deficiency, including Wolman disease and cholesteryl ester storage disease (CESD). Also known as: N-acetylgalactosamine-6-sulfatase, N-acetylgalactosamine-6-sulfate sulfatase, galactose-6-sulfatase, GALNS.
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 is a recombinant human lysosomal acid lipase (LAL) enzyme replacement therapy that restores deficient enzyme activity in patients with LAL deficiency. Used for Lysosomal acid lipase (LAL) deficiency, including Wolman disease and cholesteryl ester storage disease (CESD).
At a glance
| Generic name | BMN 110 - Every Other Week |
|---|---|
| Also known as | N-acetylgalactosamine-6-sulfatase, N-acetylgalactosamine-6-sulfate sulfatase, galactose-6-sulfatase, GALNS, enzyme replacement therapy |
| Sponsor | BioMarin Pharmaceutical |
| Drug class | Enzyme replacement therapy |
| Target | Lysosomal acid lipase (LAL) |
| Modality | Small molecule |
| Therapeutic area | Rare genetic metabolic disorder |
| Phase | Phase 3 |
Mechanism of action
BMN 110 is designed to replace the deficient lysosomal acid lipase enzyme, which normally breaks down cholesterol esters and triglycerides within lysosomes. In LAL deficiency (also known as Wolman disease or cholesteryl ester storage disease), accumulation of these lipids causes multi-organ damage. The every-other-week dosing regimen aims to maintain therapeutic enzyme levels with reduced infusion frequency compared to weekly alternatives.
Approved indications
- Lysosomal acid lipase (LAL) deficiency, including Wolman disease and cholesteryl ester storage disease (CESD)
Common side effects
- Infusion-related reactions
- Headache
- Nausea
- Fatigue
Key clinical trials
- Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) (PHASE3)
- A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) (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 110 - Every Other Week CI brief — competitive landscape report
- BMN 110 - Every Other Week updates RSS · CI watch RSS
- BioMarin Pharmaceutical portfolio CI
Frequently asked questions about BMN 110 - Every Other Week
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Related
- Drug class: All Enzyme replacement therapy drugs
- Target: All drugs targeting Lysosomal acid lipase (LAL)
- Manufacturer: BioMarin Pharmaceutical — full pipeline
- Therapeutic area: All drugs in Rare genetic metabolic disorder
- Indication: Drugs for Lysosomal acid lipase (LAL) deficiency, including Wolman disease and cholesteryl ester storage disease (CESD)
- Also known as: N-acetylgalactosamine-6-sulfatase, N-acetylgalactosamine-6-sulfate sulfatase, galactose-6-sulfatase, GALNS, enzyme replacement therapy
- Compare: BMN 110 - Every Other Week vs similar drugs
- Pricing: BMN 110 - Every Other Week cost, discount & access