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Elelyso (Taliglucerase Alfa)
Recombinant glucocerebrosidase enzyme replacement therapy that catalyzes hydrolysis of glucocerebroside to glucose and ceramide.
ELELYSO is a recombinant glucocerebrosidase enzyme replacement therapy indicated for patients 4 years and older with confirmed Type 1 Gaucher disease. The drug demonstrates nonlinear pharmacokinetics with greater-than-dose-proportional exposure increases and no significant accumulation with repeated every-other-week dosing. No contraindications are listed, and no drug interactions are documented in the label. The therapy addresses the underlying enzymatic deficiency by reducing glucocerebroside accumulation in lysosomes through mannose receptor-mediated cellular uptake.
At a glance
| Generic name | Taliglucerase Alfa |
|---|---|
| Sponsor | Pfizer Inc. |
| Drug class | Enzyme replacement therapy |
| Target | Glucocerebroside (substrate); mannose receptors on cell surface |
| Modality | Enzyme replacement |
| Therapeutic area | Rare Disease |
| Phase | FDA-approved |
Mechanism of action
Gaucher disease is an autosomal recessive lysosomal storage disorder caused by mutations in the glucocerebrosidase gene, resulting in reduced enzymatic activity. This deficiency causes accumulation of the sphingolipid glucocerebroside primarily in macrophage lysosomes, forming characteristic Gaucher cells that accumulate in the liver, spleen, and bone marrow. ELELYSO is a recombinant analog of human lysosomal glucocerebrosidase that catalyzes the hydrolysis of glucocerebroside into glucose and ceramide, reducing substrate accumulation. The drug's cellular uptake is mediated by binding of its mannose oligosaccharide chains to specific mannose receptors on the cell surface, leading to internalization and transport to lysosomes where enzymatic activity occurs.
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
- Upper respiratory infection
- Cough
- Urticaria
- Hematoma
- Fever
- Vomitting
- Pneumonia
- Labored breathing
- Snoring
- Suffocation
- Sweating
- Mosquito bite
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)
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Elelyso CI brief — competitive landscape report
- Elelyso updates RSS · CI watch RSS
- Pfizer Inc. portfolio CI