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Imiglucerase (glucocerebrosidase): Pediatric drug information

Imiglucerase (glucocerebrosidase): Pediatric drug information
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For additional information see "Imiglucerase (glucocerebrosidase): Drug information" and "Imiglucerase (glucocerebrosidase): Patient drug information"

For abbreviations, symbols, and age group definitions show table
ALERT: US Boxed 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 and after extended duration of therapy. Initiate imiglucerase in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (eg, anaphylaxis) occurs, discontinue imiglucerase 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.

Brand Names: US
  • Cerezyme
Brand Names: Canada
  • Cerezyme
Therapeutic Category
  • Enzyme, Glucocerebrosidase;
  • Gaucher's Disease, Treatment Agent
Dosing: Pediatric

Dosage guidance:

Clinical considerations: Pretreatment with antihistamines and/or corticosteroids can be considered for prevention of subsequent infusion reactions in patients with previous hypersensitivity reactions to imiglucerase or other enzyme replacement therapies. Initiate in a health care setting with appropriate medical monitoring and support measures, including CPR equipment, readily available during administration.

Gaucher disease, type 1

Gaucher disease, type 1:

Children and Adolescents: Limited data available for children <2 years:

Initial: IV: 30 to 60 units/kg/dose every 2 weeks; consider starting on higher end of range in patients with severe disease or at high risk for complications (Ref); may increase dose in patients initiated at lower end of range if response inadequate (Ref); doses >60 units/kg/dose are rarely needed (Ref).

Maintenance: Limited data available: IV: Assess calculated dose and patient growth (weight) frequently to maintain consistent dosage per kg body weight (Ref). The appropriate dose to prevent long-term complications in pediatric patients is unknown; after therapeutic goals are achieved, any dose reduction should be considered with extreme caution and at intervals no more frequent than every 6 months; minimum dose: 30 units/kg/dose every 2 weeks (Ref).

Gaucher disease, type 3

Gaucher disease, type 3: Limited data available: Note: In the management of type 3 Gaucher disease, enzyme replacement therapy is recommended to ameliorate visceral, skeletal, and hematologic manifestations; it has not been demonstrated to impact neurological manifestations.

Children and Adolescents: IV: Initial: 60 units/kg/dose every 2 weeks (Ref). Individualize dose based on patient response; doses up to 120 units/kg every 2 weeks have been described (Ref).

Dosing: Kidney Impairment: Pediatric

There are no dosage adjustments provided in the manufacturer's labeling.

Dosing: Liver Impairment: Pediatric

There are no dosage adjustments provided in the manufacturer's labeling.

Dosing: Adult

(For additional information see "Imiglucerase (glucocerebrosidase): Drug information")

Dosage guidance:

Clinical considerations: Pretreatment with antihistamines and/or corticosteroids can be considered for prevention of subsequent infusion reactions in patients with previous hypersensitivity reactions to imiglucerase or other enzyme-replacement therapies. Initiate in a health care setting with appropriate medical monitoring and support measures, including CPR equipment, readily available during administration.

Gaucher disease, type 1

Gaucher disease, type 1: IV: Initial range: 2.5 units/kg 3 times weekly, up to 60 units/kg every 2 weeks. Note: Dosage adjustments are made based on assessment and therapeutic goals. Most benefits observed with doses of 30 to 60 units/kg every 2 weeks (Ref).

Gaucher disease, type 3

Gaucher disease, type 3 (Canadian labeling; not in US labeling): IV: Initial range: 2.5 units/kg 3 times weekly, up to 60 units/kg every 2 weeks. Doses up to 120 units/kg every 2 weeks have been safely administered.

Dosing: Kidney Impairment: Adult

There are no dosage adjustments provided in the manufacturer’s labeling.

Dosing: Liver Impairment: Adult

There are no dosage adjustments provided in the manufacturer’s labeling.

Adverse Reactions

The following adverse drug reactions and incidences are derived from product labeling unless otherwise specified.

>10%: Immunologic: Development of IgG antibodies (15%)

Frequency not defined:

Cardiovascular: Tachycardia

Dermatologic: Skin rash

Gastrointestinal: Abdominal pain, diarrhea, nausea, vomiting

Hypersensitivity: Hypersensitivity reaction (including anaphylaxis, angioedema, severe hypersensitivity reaction), infusion-related reaction

Local: Burning sensation at injection site, discomfort at injection site, swelling at injection site

Nervous system: Chills, dizziness, fatigue, headache

Neuromuscular & skeletal: Back pain

Respiratory: Pneumonia, pulmonary hypertension

Miscellaneous: Fever

Contraindications

There are no known contraindications listed in the US manufacturer's labeling.

Canadian labeling: Severe hypersensitivity to imiglucerase or any component of the formulation

Warnings/Precautions

Concerns related to adverse effects:

• Antibody formation: Development of IgG antibodies has been reported in ~15% of patients and has been observed within 6 months from the onset of therapy; antibody formation is rare after 12 months of therapy; may increase risk of hypersensitivity reactions.

• Hypersensitivity reactions: Imiglucerase may cause severe hypersensitivity reactions, including anaphylaxis (sometimes life-threatening). Other reactions have included angioedema, chest discomfort, cough, cyanosis, dyspnea, flushing, hypotension, pruritus, tachycardia, and urticaria. Incidence is increased in patients with antibodies to imiglucerase. Discontinue treatment and institute appropriate therapy if hypersensitivity occurs. When retreating patients with prior hypersensitivity, administer antihistamine and/or corticosteroid premedications and decrease infusion rate.

• Infusion-related reactions: Imiglucerase may cause infusion-related reactions, including angioedema, chest discomfort, chills, fatigue, fever, hypertension, infusion-site burning/discomfort/swelling, pruritus, rash, and urticaria. Consider decreasing the infusion rate, temporarily withholding the infusion, and/or administering antihistamines, and/or antipyretics if infusion-related reactions occur.

Dosage form specific issues:

• Polysorbate 80: Some dosage forms may contain polysorbate 80 (also known as Tweens). Hypersensitivity reactions, usually a delayed reaction, have been reported following exposure to pharmaceutical products containing polysorbate 80 in certain individuals (Isaksson 2002; Lucente 2000; Shelley 1995). Thrombocytopenia, ascites, pulmonary deterioration, and renal and hepatic failure have been reported in premature neonates after receiving parenteral products containing polysorbate 80 (Alade 1986; CDC 1984). See manufacturer’s labeling.

Other warnings/precautions:

• Registry: A registry has been established and all patients with Gaucher disease, and physicians who treat Gaucher disease are encouraged to participate. Information on the International Collaborative Gaucher Group (ICGG) Gaucher Registry may be obtained at https://www.registrynxt.com, or by calling 1-800-745-4447 (ext.15500).

Warnings: Additional Pediatric Considerations

Based on postmarketing reporting, the most common reported adverse events in children 2 to 12 years include dyspnea, fever, nausea, flushing, vomiting, and coughing; for children ≥12 years and adolescents, the most common reported adverse events include headache, pruritus, and rash.

Dosage Forms: US

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Solution Reconstituted, Intravenous [preservative free]:

Cerezyme: 400 units (1 ea) [contains polysorbate 80]

Generic Equivalent Available: US

No

Pricing: US

Solution (reconstituted) (Cerezyme Intravenous)

400 unit (per each): $2,080.96

Disclaimer: A representative AWP (Average Wholesale Price) price or price range is provided as reference price only. A range is provided when more than one manufacturer's AWP price is available and uses the low and high price reported by the manufacturers to determine the range. The pricing data should be used for benchmarking purposes only, and as such should not be used alone to set or adjudicate any prices for reimbursement or purchasing functions or considered to be an exact price for a single product and/or manufacturer. Medi-Span expressly disclaims all warranties of any kind or nature, whether express or implied, and assumes no liability with respect to accuracy of price or price range data published in its solutions. In no event shall Medi-Span be liable for special, indirect, incidental, or consequential damages arising from use of price or price range data. Pricing data is updated monthly.

Dosage Forms: Canada

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Solution Reconstituted, Intravenous:

Cerezyme: 400 units (20 mL) [contains polysorbate 80]

Administration: Pediatric

Note: Monitor patients closely for infusion-related or hypersensitivity reactions. Initiate infusion in a health care setting that includes cardiopulmonary monitoring and can provide resuscitation if necessary.

Parenteral: IV: Infusion rate depends on weight; infuse diluted solution over 2 hours for patients <18 kg, and over 1 to 2 hours for patients ≥18 kg. A maximum infusion rate of 1 unit/kg/minute is described in Canadian labeling; infusion times <1 hour are not recommended (Ref). May filter diluted solution through an in-line low-protein-binding 0.2 micron filter during administration.

Rate adjustment for infusion-related reactions or hypersensitivity:

Infusion-related reactions or mild/moderate hypersensitivity reactions: Treatment should be based on the severity of the reaction and may include temporarily interrupting the infusion, decreasing the infusion rate, and/or administration of antihistamines, antipyretics, and/or corticosteroids (Ref). Consider pretreatment with antihistamines and/or corticosteroids for prevention of subsequent infusion reactions in patients with previous hypersensitivity reactions to imiglucerase or other enzyme replacement therapies; closely monitor when readministering.

Anaphylaxis or severe hypersensitivity: Discontinue therapy immediately and treat appropriately. If the decision is made to readminister after anaphylaxis, consider premedication with antihistamines and/or corticosteroids for subsequent infusions and reduction of infusion rate; appropriately trained personnel and emergency medications should be immediately available. Monitor patient closely for recurrence.

Administration: Adult

IV: Infuse over 1 to 2 hours; may filter diluted solution through an in-line, low protein-binding 0.2-micron filter during administration. The Canadian labeling recommends a maximum infusion rate of 1 unit/kg/minute. Infusion times <1 hour are not recommended (Ref). For patients who experience infusion-related reactions, reduce rate of infusion and premedicate with an antihistamine and/or corticosteroid.

Storage/Stability

Store intact vials at 2°C to 8°C (36°F to 46°F). Reconstituted solution is stable for 12 hours at 25°C (77°F) or 2°C to 8°C (36°F to 46°F). Solution diluted for infusion in NS is stable for up to 24 hours when stored at 2°C to 8°C (36°F to 46°F). Discard unused solution.

Use

Long-term enzyme replacement therapy for patients with type 1 Gaucher disease (FDA approved in ages ≥2 years and adults); has also been used as enzyme replacement therapy for patients with type 3 Gaucher disease.

Medication Safety Issues
Sound-alike/look-alike issues:

Cerezyme may be confused with Cerebyx, Ceredase

Metabolism/Transport Effects

None known.

Drug Interactions

Note: Interacting drugs may not be individually listed below if they are part of a group interaction (eg, individual drugs within “CYP3A4 Inducers [Strong]” are NOT listed). For a complete list of drug interactions by individual drug name and detailed management recommendations, use the drug interactions program

There are no known significant interactions.

Reproductive Considerations

Patients requiring treatment for type 1 Gaucher disease should continue imiglucerase while attempting to become pregnant to stabilize disease and achieve optimal health prior to pregnancy. Imiglucerase does not need to be initiated prior to conception in asymptomatic patients (Granovsky-Grisaru 2011).

Pregnancy Considerations

An increased risk of adverse pregnancy outcomes has not been associated with maternal use of imiglucerase (Raynor 2019; Zimran 2009).

Pregnancy may exacerbate existing type 1 Gaucher disease or result in new symptoms. Patients with type 1 Gaucher disease have an increased risk of spontaneous abortion if disease is not well-controlled. Adverse events, including anemia, hepatosplenomegaly, and thrombocytopenia, may be exacerbated during pregnancy (Granovsky-Grisaru 2011). Based on available information, treatment with imiglucerase can reduce the risk of adverse pregnancy outcomes (Zimran 2009).

Patients requiring treatment for type 1 Gaucher disease should continue imiglucerase during pregnancy. Treatment may be initiated during pregnancy for symptomatic patients. Dosing should be based on prepregnancy weight and adjusted if clinically indicated (Granovsky-Grisaru 2011).

Data collection to monitor pregnancy and infant outcomes following exposure to imiglucerase is ongoing. Health care providers are encouraged to enroll patients exposed to imiglucerase during pregnancy in the Gaucher Patient Registry (1-800-745-4447, extension 15500 or https://www.registrynxt.com). Patients may also enroll themselves.

Monitoring Parameters

Signs/symptoms of anaphylaxis/hypersensitivity or infusion-related reactions; IgG antibody formation periodically during the first year of treatment (Canadian labeling suggests every 3 months and at 18 months after treatment initiation).

Mechanism of Action

Imiglucerase is an analogue of glucocerebrosidase; it is produced by recombinant DNA technology using mammalian cell culture. Glucocerebrosidase is an enzyme deficient in Gaucher's disease. It is needed to catalyze the hydrolysis of glucocerebroside to glucose and ceramide.

Pharmacokinetics (Adult Data Unless Noted)

Distribution: Vd: 0.09 to 0.15 L/kg

Half-life elimination: 3.6 to 10.4 minutes

Clearance: 9.8 to 20.3 mL/minute/kg

Brand Names: International
International Brand Names by Country
For country code abbreviations (show table)

  • (AR) Argentina: Cerezyme;
  • (BE) Belgium: Cerezyme;
  • (BG) Bulgaria: Cerezyme;
  • (BR) Brazil: Cerezyme;
  • (CL) Chile: Cerezyme;
  • (CO) Colombia: Bantace | Cerezyme;
  • (CZ) Czech Republic: Cerezyme;
  • (DE) Germany: Cerezyme;
  • (EC) Ecuador: Abcertin | Cerezyme;
  • (EE) Estonia: Cerezyme;
  • (EG) Egypt: Cerezyme;
  • (FI) Finland: Cerezyme;
  • (FR) France: Cerezyme;
  • (GB) United Kingdom: Cerezyme;
  • (GR) Greece: Cerezyme;
  • (HK) Hong Kong: Cerezyme;
  • (HR) Croatia: Cerezyme;
  • (HU) Hungary: Cerezyme;
  • (IE) Ireland: Cerezyme;
  • (IT) Italy: Cerezyme;
  • (JP) Japan: Cerezyme;
  • (KR) Korea, Republic of: Abcertin | Cerezyme;
  • (KW) Kuwait: Cerezyme;
  • (LB) Lebanon: Cerezyme;
  • (LT) Lithuania: Cerezyme;
  • (MX) Mexico: Asbroder | Cerezyme;
  • (MY) Malaysia: Cerezyme;
  • (NL) Netherlands: Cerezyme;
  • (NO) Norway: Cerezyme;
  • (NZ) New Zealand: Cerezyme;
  • (PE) Peru: Abcertin | Cerezyme;
  • (PK) Pakistan: Cerezyme;
  • (PL) Poland: Cerezyme;
  • (PT) Portugal: Cerezyme;
  • (PY) Paraguay: Cerezyme;
  • (QA) Qatar: Cerezyme;
  • (RO) Romania: Cerezyme;
  • (RU) Russian Federation: Cerezyme | Glurasim;
  • (SA) Saudi Arabia: Cerezyme;
  • (SE) Sweden: Cerezyme;
  • (SG) Singapore: Cerezyme;
  • (SI) Slovenia: Cerezyme;
  • (SK) Slovakia: Cerezyme;
  • (TH) Thailand: Cerezyme;
  • (TN) Tunisia: Cerezyme;
  • (TR) Turkey: Cerezyme;
  • (TW) Taiwan: Cerezyme;
  • (UA) Ukraine: Cerezyme;
  • (ZA) South Africa: Cerezyme
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  2. Andersson HC, Charrow J, Kaplan P, et al. Individualization of long-term enzyme replacement therapy for Gaucher disease. Genet Med. 2005;7(2):105-110. [PubMed 15714077]
  3. Baldellou A, Andria G, Campbell PE, et al, "Paediatric Non-Neuronopathic Gaucher Disease: Recommendations for Treatment and Monitoring," Eur J Pediatr, 2004, 163(2):67-75. [PubMed 14677062]
  4. Centers for Disease Control (CDC). Unusual syndrome with fatalities among premature infants: association with a new intravenous vitamin E product. MMWR Morb Mortal Wkly Rep. 1984;33(14):198-199. http://www.cdc.gov/mmwr/preview/mmwrhtml/00000319.htm. [PubMed 6423951]
  5. Cerezyme (imiglucerase) [prescribing information]. Cambridge, MA: Genzyme Corporation; December 2024.
  6. Cerezyme (imiglucerase) [product monograph]. Toronto, Ontario, Canada; Sanofi-Aventis Canada Inc; May 2024.
  7. Charrow J, Andersson HC, Kaplan P, et al, "Enzyme Replacement Therapy and Monitoring for Children With Type 1 Gaucher Disease: Consensus Recommendations," J Pediatr, 2004, 144(1):112-20. [PubMed 14722528]
  8. Goker-Alpan O, Wiggs EA, Eblan MJ, et al. Cognitive outcome in treated patients with chronic neuronopathic Gaucher disease. J Pediatr. 2008;153(1):89-94. doi 10.1016/j.jpeds.2007.12.023 [PubMed 18571543]
  9. Granovsky-Grisaru S, Belmatoug N, vom Dahl S, et al. The management of pregnancy in Gaucher disease. Eur J Obstet Gynecol Reprod Biol. 2011;156(1):3-8. doi:10.1016/j.ejogrb.2010.12.024 [PubMed 21269752]
  10. Isaksson M, Jansson L. Contact allergy to Tween 80 in an inhalation suspension. Contact Dermatitis. 2002;47(5):312-313. [PubMed 12534540]
  11. Kaplan P, Baris H, De Meirleir L, et al. Revised recommendations for the management of Gaucher disease in children. Eur J Pediatr. 2013;172(4):447-458. [PubMed 22772880]
  12. Lucente P, Iorizzo M, Pazzaglia M. Contact sensitivity to Tween 80 in a child. Contact Dermatitis. 2000;43(3):172. [PubMed 10985636]
  13. Martins AM, Valadares ER, Porta G, et al, “Recommendations on Diagnosis, Treatment, and Monitoring for Gaucher Disease,” J Pediatr, 2009, 155(4 Suppl):10-8. [PubMed 19765407]
  14. Raynor L, Batista J, Carloni L, et al. Real-world outcomes in pregnant imiglucerase-treated patients with Gaucher disease: data from the global safety database and International Collaborative Gaucher Group (ICGG) Gaucher registry pregnancy sub-registry maintained by Sanofi Genzyme. Molecular Genetics and Metabolism. 2019;126(2):S122. doi:10.1016/j.ymgme.2018.12.311
  15. Refer to manufacturer's labeling.
  16. Sekijima Y, Ohashi T, Ohira S, et al. Successful pregnancy and lactation outcome in a patient With Gaucher disease receiving enzyme replacement therapy, and the subsequent distribution and excretion of imiglucerase in human breast milk. Clin Ther. 2010;32(12):2048-2052. doi:10.1016/j.clinthera.2010.11.008 [PubMed 21118740]
  17. Shelley WB, Talanin N, Shelley ED. Polysorbate 80 hypersensitivity. Lancet. 1995;345(8980):1312-1313. [PubMed 7746084]
  18. Vellodi A, Tylki-Szymanska A, Davies EH, et al. Management of neuronopathic Gaucher disease: revised recommendations. J Inherit Metab Dis. 2009;32(5):660-664. [PubMed 19655269]
  19. Zimran A, Morris E, Mengel E, et al. The female Gaucher patient: the impact of enzyme replacement therapy around key reproductive events (menstruation, pregnancy and menopause). Blood Cells Mol Dis. 2009;43(3):264-288. doi:10.1016/j.bcmd.2009.04.003 [PubMed 19502088]
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