Sebelipase alfa

Overview

The lysosomal acid lipase (LAL) enzyme is found in lysosomes and is primarily responsible for the metabolism of lipids, and its absence or deficiency results in the accumulation of lipids in various organs. This lipid accumulation can lead to end-organ damage in the form of liver dysfunction or malabsorption secondary to intestinal dysfunction. In addition, patients with LAL deficiency typically develop dyslipidemia, which itself contributes to a number of adverse cardiovascular outcomes. Sebelipase alfa is a recombinant form LAL approved for the treatment of LAL deficiency. It was first approved by both the FDA and EMA in 2015 and is marketed under the brand name Kanuma (Alexion Pharmaceuticals).

Indication

Sebelipase alfa is a hydrolytic lysosomal cholesteryl ester and triacylglycerol-specific enzyme indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency.

Associated Conditions

Lysosomal Acid Lipase Deficiency

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency	2015/03/03	NCT02376751	N/A	NO_LONGER_AVAILABLE	Alexion Pharmaceuticals, Inc.
Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency	2014/07/18	NCT02193867	Phase 2	Terminated	Alexion Pharmaceuticals, Inc.
Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency	2014/04/14	NCT02112994	Phase 2	Completed	Alexion Pharmaceuticals, Inc.
Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency	2012/12/28	NCT01757184	Phase 3	Completed	Alexion Pharmaceuticals, Inc.
Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency	2011/12/08	NCT01488097	Phase 2	Completed	Alexion Pharmaceuticals, Inc.

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
KANUMA	Alexion Pharmaceuticals, Inc.	25682-007	INTRAVENOUS	2 mg in 1 mL	11/24/2021

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
Kanuma	Alexion Europe SAS,103-105 rue Anatole France,92300 Levallois-Perret,France	Authorised	8/28/2015

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
KANUMA CONCENTRATE FOR SOLUTION FOR INFUSION 20MG/10ML	N/A	ASTRAZENECA HONG KONG LIMITED	N/A	N/A	6/20/2025

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
KANUMA sebelipase alfa rce 2 mg/mL injection intravenous infusion vial	274498	Alexion Pharmaceuticals Australasia Pty Ltd	Medicine	A	5/18/2017

Health Canada Drug Approvals

Approved Product	Company	DIN	Dosage Form	Strength	Market Date
KANUMA	Alexion Pharma GmbH	02469596	Solution - Intravenous	2 MG / ML	4/5/2018

CIMA AEMPS Drug Approvals

Approved Product	Company	Registration Number	Pharmaceutical Form	Prescription Type	Status
KANUMA 2 MG/ML CONCENTRADO PARA SOLUCION PARA PERFUSION	Alexion Europe Sas	1151033001	CONCENTRADO PARA SOLUCIÓN PARA PERFUSIÓN	Uso Hospitalario	Commercialized

Philippines FDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Philippines FDA approvals found for this drug.

Saudi SFDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Saudi SFDA approvals found for this drug.

Malaysia NPRA Drug Approvals

Approved Product	Company	Registration Number	Dosage Form	Strength	Approval Date
No Malaysia NPRA approvals found for this drug.

UK EMC Drug Information

Medicine Name	MA Holder	MA Number	Pharmaceutical Form	Active Ingredient	Authorization Date
No UK EMC drug information found for this drug.

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NICE Rejects Alexion's Kanuma for Ultra-Rare LAL-D Treatment, Citing High Costs

8 years ago

• NICE has rejected Alexion's Kanuma (sebelipase alfa) for lysosomal acid lipase deficiency treatment, marking their second ultra-rare disease drug rejection in eight days. • At £491,992 per patient annually, Kanuma becomes NICE's most expensive drug review to date, with the agency citing uncertain long-term benefits despite confidential discount offers. • The decision affects approximately 25 patients in England with LAL-D, particularly impacting infants under six months who have no alternative treatment options.

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