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BMN 165 — Competitive Intelligence Brief

BMN 165 (BMN 165) competitive landscape: 2 comparators, recent regulatory actions, upcoming PDUFA, patent timeline. Class: Gene therapy (AAV-based). Area: Rare Genetic Disorder / Lysosomal Storage Disease.

phase 3 Gene therapy (AAV-based) GALNS gene (N-acetylgalactosamine-6-sulfatase) Rare Genetic Disorder / Lysosomal Storage Disease Small molecule Live · refreshed every 30 min

Target snapshot

BMN 165 (BMN 165) — BioMarin Pharmaceutical. 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).

Comparator set (2 drugs)

Auto-detected by same drug class / molecular target / therapeutic area, ranked by phase + recency. Override with ?vs=slug1,slug2,….

DrugGenericSponsorPhaseClassTargetApproval / PDUFA
BMN 165 TARGET BMN 165 BioMarin Pharmaceutical phase 3 Gene therapy (AAV-based) GALNS gene (N-acetylgalactosamine-6-sulfatase)
CAM-101 CAM-101 Cambium Bio Limited phase 3 Gene therapy (AAV-based) CDKN1C gene / p57 protein
BMN 110 BMN 110 BioMarin Pharmaceutical phase 3 Gene therapy (AAV-based) HEXA gene

Recent regulatory actions (last 90 days)

No regulatory actions in the last 90 days for this set.

Upcoming PDUFA dates (next 180 days)

No PDUFA dates in the next 180 days for this set.

Patent timeline (forward window)

No upcoming patent expiries for this set.

Sponsor landscape (Gene therapy (AAV-based) class)

  1. BioMarin Pharmaceutical · 2 drugs in this class
  2. Cambium Bio Limited · 1 drug in this class

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Cite this brief

Drug Landscape (2026). BMN 165 — Competitive Intelligence Brief. https://druglandscape.com/ci/bmn-165. Accessed 2026-05-13.

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