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BMN 110 - Weekly
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 - Weekly |
|---|---|
| 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 cholesteryl esters and triglycerides within lysosomes. In LAL deficiency (also known as Wolman disease or cholesteryl ester storage disease), accumulation of these lipids causes progressive organ damage. The weekly formulation aims to provide sustained enzyme replacement with less frequent dosing than prior formulations.
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)
- Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome) (PHASE2)
- Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome (PHASE2)
- Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation (PHASE2)
- A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) (PHASE3)
- A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA (PHASE1, PHASE2)
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 - Weekly CI brief — competitive landscape report
- BMN 110 - Weekly updates RSS · CI watch RSS
- BioMarin Pharmaceutical portfolio CI