Last reviewed 2026-05-27 · How we verify

TALIGLUCERASE ALFA

TALIGLUCERASE ALFA is a Hydrolytic Lysosomal Glucocerebroside-specific Enzyme [EPC] drug. It is currently FDA-approved (first approved 2012) for Type 1 Gaucher Disease.

ELELYSO replaces deficient glucocerebrosidase, reducing glucocerebroside accumulation in Gaucher cells.

Taliglucerase alfa is a plant cell-expressed recombinant glucocerebrosidase enzyme used to treat Gaucher Disease. It works by hydrolyzing glucocerebroside, a compound that accumulates in the cells of individuals with Gaucher Disease.

At a glance

Mechanism of action

ELELYSO is a recombinant form of the enzyme glucocerebrosidase, which is deficient in patients with Gaucher disease. By replacing this enzyme, ELELYSO helps break down glucocerebroside, preventing its buildup in the lysosomes of macrophages, thus reducing the formation of Gaucher cells in organs like the liver, spleen, and bone marrow.

Approved indications

• Type 1 Gaucher Disease

Boxed warnings

• WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life‑threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate ELELYSO in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue ELELYSO and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life‑threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur [see Warnings and Precautions (5.1) ] . WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS See full prescribing information for complete boxed warning. • Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. ( 5.1 ) • Initiate ELELYSO in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. ( 5.1 ) • If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue ELELYSO and immediately initiate appropriate medical treatment, including use of epinephrine. ( 5.1 )

Common side effects

• Headache
• Arthralgia
• Fatigue
• Nausea
• Dizziness
• Abdominal pain
• Pruritus
• Flushing
• Vomiting
• Urticaria
• Vomiting
• Arthralgia

Key clinical trials

• A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease (PHASE4)
• An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3 (PHASE2, PHASE3)
• A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease (PHASE3)
• Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs (NA)
• Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease (PHASE4)
• A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease (PHASE4)
• Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial (PHASE3)
• Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase (PHASE3)

Primary sources

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Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• TALIGLUCERASE ALFA CI brief — competitive landscape report
• TALIGLUCERASE ALFA updates RSS · CI watch RSS

Frequently asked questions about TALIGLUCERASE ALFA

Related

• Drug class: All Hydrolytic Lysosomal Glucocerebroside-specific Enzyme [EPC] drugs
• Target: All drugs targeting glucocerebrosidase
• Indication: Drugs for Type 1 Gaucher Disease

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing