Taliglucerase Alfa

Gaucher disease is an autosomal recessive disorder caused by mutations in the human glucocerebrosidase gene, which results in a reduced activity of the lysosomal enzyme glucocerebrosidase. Glucocerebrosidase catalyzes the conversion of the sphingolipid glucocerebroside into glucose and ceramide.

• ELELYSO is indicated for the treatment of patients 4 years of age and older with a confirmed diagnosis of Type 1 Gaucher disease. ELELYSO is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for the treatment of patients 4 years and older with a confirmed diagnosis of Type 1 Gaucher disease ( 1 ).

• None.contraindicated

Recommendations Prior to ELELYSO Treatment ( 2.1 ): • Administration of ELELYSO should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. Recommended Dosage in Patients 4 Years and Older ( 2.2 ): • Treatment-naïve : 60 units/kg administered every other week as a 60- to 120-minute intravenous infusion. • Patients switching from imiglucerase : Initiate ELELYSO intravenous treatment (60- to 120-minute infusion) with the same units/kg imiglucerase dosage and subsequently administer ELELYSO every other week. Dosage adjustments can be based on achievement and maintenance of each patient’s therapeutic goals. Preparation and Administration ( 2.3, 2.4, 2.5 ): • Reconstitute, dilute and administer under the supervision of a healthcare professional. • See Full Prescribing Information for complete instructions. 2.1 Recommendations Prior to ELELYSO Treatment Administration of ELELYSO should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis [see Warnings and Precautions (5.1) ] . Initiate ELELYSO in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment [see Warnings and Precautions (5.1) ] . To reduce the risk of hypersensitivity reactions, consider pretreatment with antihistamines and/or corticosteroids [see Warnings and Precautions (5.1) ] .

See boxed warning ( 5.1 ) 5.1 Hypersensitivity Reactions Including Anaphylaxis Life‑threatening h ypersensitivity reactions, including anaphylaxis, have occurred in patients treated with enzyme replacement therapies, including ELELYSO . In clinical trials, (patients were not routinely pretreated with antihistamines and/or corticosteroids prior to ELELYSO infusions during the clinical trials): • 2 of 72 (3%) ELELYSO-treated patients experienced signs and symptoms consistent with anaphylaxis including urticaria, hypotension, flushing, wheezing, chest tightness, nausea, vomiting, and dizziness. These reactions occurred during ELELYSO infusion. • 21 of 72 (29%) ELELYSO-treated patients experienced hypersensitivity reactions, including the 2 ELELYSO‑treated patients who experienced signs and symptoms consistent with anaphylaxis. Signs and symptoms of these hypersensitivity reactions included pruritus, angioedema, flushing, erythema, rash, nausea, vomiting, cough, chest tightness, and throat irritation. These reactions occurred during ELELYSO infusion and up to 3 hours after the start of infusion [see Adverse Reactions (6.1) ] . Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Administration of ELELYSO should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. Initiate ELELYSO in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment.

The most common adverse reactions are: • Treatment-Naïve Adults (≥5%): headache, arthralgia, fatigue, nausea, dizziness, abdominal pain, pruritus, flushing, vomiting, urticaria ( 6.1 ). • Patients who Switched from Imiglucerase, after 9 Months on Treatment (≥10%): arthralgia, headache, pain in extremity ( 6.1 ). To report SUSPECTED ADVERSE REACTIONS, contact Pfizer Inc. at 1-800-438-1985 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. Adverse Reactions from Clinical Trials of ELELYSO as Initial Therapy • Clinical Trial in Adult Patients The safety of ELELYSO at dosages of either 30 units/kg (n=16) (50% of the recommended dosage) [see Dosage and Administration (2.1) ] or 60 units/kg (n=16) administered intravenously every other week was assessed in 32 adult treatment-naïve patients (aged 19 to 74 years) with Type 1 Gaucher disease in a 9-month double-blind, randomized clinical trial (Trial 1) [see Clinical Studies (14) ] . Table 1 presents the adverse reactions that occurred in these ELELYSO-treated patients.

8.1 Pregnancy Risk Summary Available data, including data from a product registry and pharmacovigilance reports, have not identified a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes in pregnant women exposed to ELELYSO. Additionally, there are risks associated with untreated symptomatic Type I Gaucher disease in pregnant women ( see Clinical Considerations ). In animal reproduction studies when pregnant rats and rabbits were administered taliglucerase alfa at intravenous doses up to 5 times the recommended human dose (RHD), there was no evidence of embryo-fetal toxicity ( see Data ). The background risk of major birth defects and miscarriage for the indicated population is unknown. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively. Clinical Considerations Disease-Associated Maternal and/or Embryo/Fetal Risk Women with Type 1 Gaucher disease have an increased risk of spontaneous abortion if disease symptoms are not treated and controlled pre-conception and during a pregnancy. Pregnancy may exacerbate existing Type 1 Gaucher disease symptoms or result in new disease manifestations.

Metabolism

Pharmacokinetics of taliglucerase alfa were evaluated in 38 patients (29 adult and 9 pediatric patients) who received intravenous infusions of ELELYSO 30 units/kg (50% of the recommended dosage) or 60 units/kg (the recommended dosage) [see Dosage and Administration (2.1) ] every other week. The pharmacokinetic parameters in adult and pediatric patients are summarized in Table 3.

• May 1, 2012BLABLA022458Pfizer

1. 1Fatigue6912%
2. 2Off Label Use488.7%
3. 3Arthralgia468.3%
4. 4Headache458.1%
5. 5Malaise458.1%
6. 6Pain448.0%
7. 7Pain In Extremity448.0%
8. 8Pyrexia448.0%
9. 9Dyspnoea397.1%
10. 10Inappropriate Schedule Of Product Administration386.9%
11. 11Nausea366.5%
12. 12Back Pain346.1%
13. 13Infusion Related Reaction346.1%
14. 14Cough305.4%
15. 15Abdominal Pain295.2%

The 10 most recently updated of 12 ClinicalTrials.gov registrations naming Taliglucerase Alfa as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease
  Completed · Phase 4 · Interventional · 14 enrolled · Ari Zimran
  NCT04002830updated 2024-12-17
• An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3
  Withdrawn · Phase 2 · Phase 3 · Interventional · 0 enrolled · AVROBIO
  NCT05815004updated 2023-08-09
• A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease
  Completed · Phase 3 · Interventional · 19 enrolled · Pfizer
  NCT01422187updated 2023-04-19
• Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs
  Completed · Interventional · 30 enrolled · Shaare Zedek Medical Center
  NCT04353466updated 2022-10-28
• Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease
  Withdrawn · Phase 4 · Interventional · 0 enrolled · Pfizer
  NCT03021941updated 2019-09-24
• A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
  Completed · Phase 4 · Interventional · 11 enrolled · Pfizer
  NCT01132690updated 2018-10-05
• Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
  Completed · Phase 3 · Interventional · 45 enrolled · Pfizer
  NCT00705939updated 2018-10-04
• A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
  Completed · Phase 3 · Interventional · 32 enrolled · Pfizer
  NCT00376168updated 2018-10-04
• Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase
  Completed · Phase 3 · Interventional · 31 enrolled · Pfizer
  NCT00712348updated 2018-10-04
• Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
  No longer available · Expanded access · Pfizer
  NCT00962260updated 2018-09-07

Frequently asked questions

How does Taliglucerase Alfa work?

Gaucher disease is an autosomal recessive disorder caused by mutations in the human glucocerebrosidase gene, which results in a reduced activity of the lysosomal enzyme glucocerebrosidase. Glucocerebrosidase catalyzes the conversion of the sphingolipid glucocerebroside into glucose and ceramide.

What is Taliglucerase Alfa used for?

According to FDA labeling, Taliglucerase Alfa carries indications including: ELELYSO is indicated for the treatment of patients 4 years of age and older with a confirmed diagnosis of Type 1 Gaucher disease. ELELYSO is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for the treatment of patients 4 years and older with a confirmed diagnosis of Type 1 Gaucher disease ( 1 ).. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Taliglucerase Alfa?

Taliglucerase Alfa is classified as Enzymes, Hydrolytic Lysosomal Glucocerebroside-specific Enzyme, Enzymatic Activity, Lipid Metabolism Alteration.

What are the brand names for Taliglucerase Alfa?

Taliglucerase Alfa is marketed under brand names including Elelyso.

What are the contraindications for Taliglucerase Alfa?

Taliglucerase Alfa labeling lists contraindications including: None.. Always consult the full prescribing information and a clinician.