Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Sebelipase alfa/kg · 1 trial · 3 indications
Safety and tolerability of sebelipase alfa was primarily assessed by monitoring the number of participants reporting treatment-emergent adverse events (TEAEs), including serious adverse events, and infusion-related reactions (IRRs). The number of participants who discontinued from the study due to a TEAE is also presented. An IRR was defined as any adverse event that occurred between the start of the infusion and 4 hours after completion of the infusion and was assessed by the Investigator as at least possibly related to study drug. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
| Arm | Type | Description |
|---|---|---|
| Sebelipase alfa 0.35 mg/kg | EXPERIMENTAL | Cohort 1: Participants were administered once weekly (qw) infusions of 0.35 mg/kg sebelipase alfa. |
| Sebelipase alfa 1 mg/kg | EXPERIMENTAL | Cohort 2: Participants were administered qw infusions of 1 mg/kg sebelipase alfa. |
| Sebelipase alfa 3 mg/kg | EXPERIMENTAL | Cohort 3: Participants were administered qw infusions of 3 mg/kg sebelipase alfa. |
| Name | Type | Description |
|---|---|---|
| Sebelipase alfa 0.35 mg/kg | DRUG | Sebelipase alfa is a recombinant human lysosomal acid lipase. |
| Sebelipase alfa 1 mg/kg | DRUG | Sebelipase alfa is a recombinant human lysosomal acid lipase. |
| Sebelipase alfa 3 mg/kg | DRUG | Sebelipase alfa is a recombinant human lysosomal acid lipase. |
Inclusion Criteria: * Male or female participants ≥ 18 and ≤ 65 years of age * Documented decreased LAL activity * Evidence of liver involvement Exclusion Criteria: * Clinically significant concurrent disease, serious inter-current illness, concomitant medications or other extenuating circumstanc...
Sebelipase Alfa is an investigational enzyme replacement therapy being developed for lysosomal acid lipase deficiency, a rare genetic condition. It is studied in patients with cholesterol ester storage disease (CESD), Wolman disease, and related forms of LAL deficiency. The drug is administered to address the underlying enzyme deficiency in these conditions.
Sebelipase Alfa is developed by AstraZeneca PLC, which trades under the ticker AZN. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with lysosomal acid lipase deficiency, including those with cholesterol ester storage disease.
Sebelipase Alfa has completed Phase 3 clinical development. The pivotal Phase 3 trial, known as ARISE (NCT01757184), enrolled 66 participants with lysosomal acid lipase deficiency. The drug remains investigational and is not yet approved by regulatory authorities.
Sebelipase Alfa has completed four clinical trials. These include a Phase 1 study (NCT01307098) in adults, a Phase 2 study (NCT01371825) in children with growth failure, the Phase 3 ARISE trial (NCT01757184), and a Phase 2 study (NCT02112994) in patients as young as 8 months.
Sebelipase Alfa is designed to replace the deficient lysosomal acid lipase enzyme in patients with LAL deficiency. By providing the missing enzyme, the drug aims to restore the breakdown of cholesteryl esters and triglycerides within lysosomes, addressing the underlying metabolic defect in conditions like cholesterol ester storage disease.
Yes, Sebelipase Alfa was previously known as SBC-102. The Phase 1 clinical trial NCT01307098 used the SBC-102 designation in its title, confirming that both names refer to the same investigational drug for lysosomal acid lipase deficiency.