Sebelipase alfa 0.35 mg/kg

Phase 2

Phase 2

Development Phase

Upcoming Catalysts

Historical Events

Regulatory Approvals

No catalyst events have been recorded for this drug yet.

Competitive Landscape

0 competitors in Cholesterol Ester Storage Disease(CESD)

Drug Information

Target Indication

Cholesterol Ester Storage Disease(CESD)

Clinical Trial

Last updated: 5/2/2026

Patent & Exclusivity

No patent data available. Pre-approval drugs may not have Orange Book listings.

Regulatory Approvals

EMA8/28/2015

Kanuma

Kanuma is indicated for long-term enzyme replacement therapy (ERT) in patients of all ages with lysosomal acid lipase (LAL) deficiency.

OrphanAccelerated

Drug Science

Mechanism of action & safety profile

Developer

AZN

AstraZeneca PLC

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