Kanuma
An intravenous enzyme replacement therapy used to treat lysosomal acid lipase deficiency.
Where Sebelipase Alfa is approved
All regulators →Of the three regulators tracked here, the first to approve Sebelipase Alfa was the EU, on 28 Aug 2015. It is approved in 3 of the 3 regulators tracked here.
From the regulators' own registers: FDA Drugs@FDA, the EMA medicines list and Health Canada's Notice of Compliance database. Dates are the first listed approval of a product containing only this substance; combination products and nationally authorised EU medicines are not counted.
Sebelipase Alfa patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Sebelipase Alfa, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- Kanuma for treatment of lysosomal acid lipase deficiency: until 1 Sep 2027 (includes 24 months' paediatric extension). Union Register
Reproduced from the FDA Orange Book and Purple Book (checked 5 Oct 2026) and the European Commission's Union Register of medicinal products (CC BY 4.0; the EU expiry date is calculated exactly as the Commission's register displays it). Listed patents and dates are as submitted to or published by the regulator and may not reflect litigation, invalidation, settlements, lapsed fees or later extensions. This is not legal advice or a freedom-to-operate opinion, and it does not say when a generic or biosimilar will launch.
How Sebelipase Alfa works
All drug targets →- Cholesteryl ester storage diseasePhase 3
- Growth delayPhase 2/3
- Liver failurePhase 2
- Decreased liver functionPhase 1/2
- Liver disorderPhase 1/2
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL3039537, CC BY-SA 3.0. The Guide to Pharmacology has no record of this medicine, so its targets rest on ChEMBL alone. Trial phases are not approvals: approved uses are shown from the regulators' own records where we hold them.
NICE appraisals of Sebelipase Alfa
All NICE appraisals →NICE, which decides whether the NHS in England should fund new medicines, has published 1 technology appraisal of Sebelipase Alfa. 1 is current.
| Appraisal | NICE recommendation | Published | Status |
|---|---|---|---|
| Sebelipase alfa for treating lysosomal acid lipase deficiency that is not Wolman disease (terminated appraisal) NICE TA961 | Terminated (no company submission) | 28 Mar 2024 | Current |
NICE decisions apply to the NHS in England. In Ireland the HSE decides on reimbursement, advised by the National Centre for Pharmacoeconomics. Titles, categories and dates link to the guidance on nice.org.uk. © NICE 2026 technology appraisal guidance. Available from www.nice.org.uk/guidance. All rights reserved. Subject to Notice of rights. NICE guidance is prepared for the National Health Service in England. All NICE guidance is subject to regular review and may be updated or withdrawn. NICE accepts no responsibility for the use of its content in this product/publication.
Patient leaflets for Sebelipase Alfa
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- EUEuropean Union: KanumaEMA product information: package leaflet and summary of product characteristics in every EU language.
- UKUnited Kingdom: Search MHRA ProductsPatient information leaflets and SmPCs for UK-licensed products.
- USUnited States: Search DailyMedFDA-approved labels, including patient and medication guides.
Leaflets differ between brands and countries; always read the one that comes with your own medicine. This is regulatory information, not medical advice.
Reference identifiers for Sebelipase Alfa
- ATC code
- A16AB14
- ChEMBL
- CHEMBL3039537
- DrugBank
- DB11563
- CAS number
- 1276027-63-4
- FDA UNII
- K4YTU42T8G
- Wikidata
- Q21789862
- Wikipedia
- Sebelipase alfa
Codes that identify this medicine in other databases, from Wikidata (CC0), matched by its ChEMBL ID. The ATC code is the WHO classification of what the medicine is used for.
What Kanuma is used for
Sebelipase Alfa (Kanuma) is an intravenous enzyme replacement therapy used to treat lysosomal acid lipase deficiency. It belongs to the Lysosomal acid lipase replacement class.
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How this page is built
The facts on this page are pulled directly from official U.S. FDA datasets — they are not written from memory. Each field below names the dataset it came from, so you can check it yourself.
- FDA Drug Label (openFDA) ↗ Indications, mechanism of action and pharmacologic class
- Drugs@FDA ↗ First FDA approval date and the company holding the application
- FDA Drug Shortage Database ↗ Current US supply / shortage status
Plain-English summaries and drug-class explainers are written and reviewed by Sreepriya Prasannan (MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany). Data is retrieved automatically from the sources above and cross-checked with AI-assisted verification (Anthropic's Claude) — brand and generic names are matched against the exact FDA product record so that a combination product or a different formulation cannot be mistaken for the drug on this page. An editor reviews the result before publication. We describe this in full in our editorial standards and corrections policy. The FDA data on this page was last retrieved on 5 Oct 2026. How every register is built: methodology · fixes we have made: corrections log.