ReviewDrugs2015
Sebelipase alfa: first global approval.
Review in Drugs, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
13 citing papers in PubMed, 36 citations in OpenAlex.
- Recombinant Protein Drugs: A 2025 Update.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2026Review
- Liver-specific gene therapy based on self-complementary adeno-associated virus for lysosomal acid lipase deficiency.Frontiers in pharmacology · 2026Article
- Overview of pediatric and adult lysosomal acid lipase deficiency: expert recommendations from a Gulf cooperation council working group.Orphanet journal of rare diseases · 2025Review
- Sebelipase Alfa Improves Aminotransferase Levels in Lysosomal Acid Lipase Deficiency: Data From an International Registry.Liver international : official journal of the International Association for the Study of the Liver · 2025Observational
- Evaluation of 73 Enlisted Patients for Liver Transplant with Unknown Etiology Reveals a Late-Diagnosed Case of Lysosomal Acid Lipase Deficiency.International journal of molecular sciences · 2024Article
- Perspectives in Genome-Editing Techniques for Livestock.Animals : an open access journal from MDPI · 2023Review
- Trends and Perspectives of Biological Drug Approvals by the FDA: A Review from 2015 to 2021.Biomedicines · 2022Review
- Therapeutic efficacy of rscAAVrh74.miniCMV.Molecular therapy. Methods & clinical development · 2022Article
- 15-Year progression to liver cancer in the lack of treatment for lysosomal acid lipase deficiency: A case report.Medicine · 2022Article
- Towards progressive regulatory approaches for agricultural applications of animal biotechnology.Transgenic research · 2022Review
- The role of sebelipase alfa in the treatment of lysosomal acid lipase deficiency.Therapeutic advances in gastroenterology · 2017Review
- Computational Approaches for the Discovery of Human Proteasome Inhibitors: An Overview.Molecules (Basel, Switzerland) · 2016Review
- Novel treatment options for lysosomal acid lipase deficiency: critical appraisal of sebelipase alfa.The application of clinical genetics · 2016Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Sebelipase alfa (Kanuma™) is a recombinant human lysosomal acid lipase (LAL) developed by Synageva BioPharma Corp. (now Alexion Pharmaceuticals, Inc.) for long-term enzyme replacement therapy in patients with LAL deficiency. The agent, administered by intravenous infusion once weekly or once every other week, acts to replace the deficient enzyme activity in patients with LAL deficiency, reducing lysosomal lipid accumulation, and thereby improving disease-related abnormalities such as dyslipidaemia and liver abnormalities. Sebelipase alfa received its first global approval, in the EU, in August 2015 for long-term enzyme replacement therapy in patients of all ages with LAL deficiency. Regulatory submissions have also been filed in the USA, Mexico and Japan for use in this indication. This article summarizes the milestones in the development of sebelipase alfa leading to this first approval for the treatment of LAL deficiency.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.