Drug intelligence / Profile preview

placebo + rhASB

Development stage: Unknown
Lead developer: BioMarin Pharmaceutical
Modality: Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
Administration: Intravenous

Overview

This refers to a clinical trial design where patients initially received placebo and then switched to rhASB (galsulfase), an enzyme replacement therapy for the treatment of mucopolysaccharidosis VI (MPS VI). rhASB is a recombinant human arylsulfatase B enzyme that helps break down glycosaminoglycans (GAGs) that accumulate in patients with MPS VI. In the Phase 3 extension study, patients who initially received placebo and then switched to rhASB for 24 weeks showed improvements in endurance measures and reduction in urinary GAG levels.

Targets

ARSB (N-acetylgalactosamine 4-sulfatase)

Beyond the preview

Go deeper on placebo + rhASB.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Clinical trials

Follow clinical development from study design and recruitment through results.

Trial phase
Status
Readouts

Indications & development

Explore development by indication, patient population, and geography.

Indications
Development status
Countries

Licensing & deals

Trace asset ownership, licensing agreements, and commercial partnerships.

Partners
Deal terms
Milestones

Patents & exclusivity

Explore the patent landscape and regulatory exclusivity around an asset.

Patents
Expiration dates
Exclusivity

Competitive landscape

Compare development programs by target, modality, and indication.

Competing assets
Targets
Development stage

Research & analysis

Connect source evidence and development news to your research questions.

Publications
News
Analysis

Bring the full picture into focus.

See how Gosset can support your research on placebo + rhASB.

Explore the full profile