Sebelipase Alfa
Lysosomal Acid Lipase Deficiency
Floatz Rating
B62/100
Confidence
Indicative
Sponsor
Alexion Pharmaceuticals, Inc.
Modality
protein_peptide
Development Phase
Phase 2/3
Status
Dormant
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
47Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT02193867CT.gov Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency | — | Terminated | — | — | — |
NCT01371825CT.gov Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency | — | Completed | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95Indicative
Detailed axis rationale is planned and will be published soon.
Same indication · Lysosomal Acid Lipase Deficiency
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Sebelipase Alfa (this asset) | Alexion Pharmaceuticals, Inc. | P2/3 | B · 62 |
Other indications for Sebelipase Alfa
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Wolman Disease | — | P2/3 | B · 58 |
Scientific Foundation
Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR
Planned for methodology v0.2.
Development Feasibility
How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR
Planned for methodology v0.2.
Commercial Opportunity
Commercial prize: addressable population, unmet need, and the value case for the indication.
NR
Planned for methodology v0.2.
IP & Exclusivity
Exclusivity position, covering patent protection and freedom-to-operate runway.
NR
Planned for methodology v0.2.
Manufacturing & Supply
Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR
Planned for methodology v0.2.
Citation
Floatz Terminal. Sebelipase Alfa in Lysosomal Acid Lipase Deficiency. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026. Accessed July 14, 2026. https://terminal.floatz.ai/assets/sebelipase-alfa-lysosomal-acid-lipase-deficiency
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