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BMN 165
BMN 165 is a gene therapy that delivers a functional copy of the GALNS gene to restore enzyme activity deficient in mucopolysaccharidosis IVA (Morquio A syndrome).
BMN 165 is a gene therapy that delivers a functional copy of the GALNS gene to restore enzyme activity deficient in mucopolysaccharidosis IVA (Morquio A syndrome). Used for Mucopolysaccharidosis IVA (Morquio A syndrome).
At a glance
| Generic name | BMN 165 |
|---|---|
| Also known as | rAvPAL-PEG, Pegvaliase |
| Sponsor | BioMarin Pharmaceutical |
| Drug class | Gene therapy (AAV-based) |
| Target | GALNS gene (N-acetylgalactosamine-6-sulfatase) |
| Modality | Small molecule |
| Therapeutic area | Rare Genetic Disorder / Lysosomal Storage Disease |
| Phase | Phase 3 |
Mechanism of action
BMN 165 uses an adeno-associated virus (AAV) vector to deliver the galactokinase (GALNS) gene into patient cells, enabling production of the missing or deficient N-acetylgalactosamine-6-sulfatase enzyme. This enzyme restores the ability to break down keratan sulfate and chondroitin-6-sulfate, reducing pathological accumulation of these glycosaminoglycans in tissues and halting or slowing disease progression.
Approved indications
- Mucopolysaccharidosis IVA (Morquio A syndrome)
Common side effects
- Elevated liver transaminases
- Immune response/antibody formation
- Injection site reactions
Key clinical trials
- Immune Modulation During Palynziq® Treatment in Adults (IMPALA) (PHASE4)
- Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria (PHASE3)
- Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU (PHASE3)
- A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
- A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks (PHASE2)
- An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165 (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 165 CI brief — competitive landscape report
- BMN 165 updates RSS · CI watch RSS
- BioMarin Pharmaceutical portfolio CI