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Galsulfase (recombinant human N-acetylgalactosamine-4 sulfatase): Pediatric drug information

Galsulfase (recombinant human N-acetylgalactosamine-4 sulfatase): Pediatric drug information
(For additional information see "Galsulfase (recombinant human N-acetylgalactosamine-4 sulfatase): Drug information" and see "Galsulfase (recombinant human N-acetylgalactosamine-4 sulfatase): Patient drug information")

For abbreviations, symbols, and age group definitions used in Lexicomp (show table)
Brand Names: US
  • Naglazyme
Brand Names: Canada
  • Naglazyme
Therapeutic Category
  • Enzyme
Dosing: Pediatric
Mucopolysaccharidosis VI

Mucopolysaccharidosis (MPS) VI: Note: Premedicate with antihistamines with or without antipyretics 30 to 60 minutes prior to infusion.

Infants: Limited data available: IV: 1 or 2 mg/kg/dose once weekly has been used in four infants (3 to 12 months); similar safety results to those of older patients were reported (Ref).

Children and Adolescents: IV: 1 mg/kg/dose once weekly

Dosing: Kidney Impairment: Pediatric

Children and Adolescents: There are no dosage adjustments provided in the manufacturer's labeling.

Dosing: Hepatic Impairment: Pediatric

Children and Adolescents: There are no dosage adjustments provided in the manufacturer's labeling.

Dosing: Adult

(For additional information see "Galsulfase (recombinant human N-acetylgalactosamine-4 sulfatase): Drug information")

Note: Premedicate with antihistamines (with/without antipyretics) 30 to 60 minutes prior to the start of the infusion.

Mucopolysaccharidosis VI

Mucopolysaccharidosis VI (MPS VI): IV: 1 mg/kg/dose once weekly.

Dosing: Kidney Impairment: Adult

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

Dosing: Hepatic 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. Adverse reactions reported in children, adolescents, and adults.

>10%:

Cardiovascular: Chest pain (16%), hypertension (11%)

Dermatologic: Skin rash (21%)

Gastrointestinal: Abdominal pain (47%), gastroenteritis (11%), umbilical hernia (11%)

Hypersensitivity: Infusion-related reaction (56%)

Immunologic: Antibody development (98%)

Nervous system: Absent reflexes (11%), chills (21%), malaise (11%), pain (32%)

Neuromuscular & skeletal: Arthralgia (42%)

Ophthalmic: Conjunctivitis (21%), corneal opacity (11%)

Otic: Auditory impairment (11%), otalgia (42%)

Respiratory: Dyspnea (21%), nasal congestion (11%), pharyngitis (11%)

Frequency not defined:

Hypersensitivity: Angioedema, nonimmune anaphylaxis, severe hypersensitivity reaction, type III hypersensitivity reaction (including membranous glomerulonephritis)

Respiratory: Apnea, laryngeal edema, respiratory distress

Postmarketing:

Cardiovascular: Bradycardia, hypotension, shock, tachycardia

Dermatologic: Erythema of skin, pallor

Hematologic & oncologic: Thrombocytopenia

Hypersensitivity: Anaphylaxis

Nervous system: Paresthesia

Renal: Kidney disease (membranous)

Respiratory: Bronchospasm, cyanosis, hypoxia, respiratory failure, tachypnea

Miscellaneous: Fever

Contraindications

There are no contraindications listed in the manufacturer's labeling

Warnings/Precautions

Concerns related to adverse effects:

• Hypersensitivity: Severe hypersensitivity reactions, including anaphylaxis, severe allergic reactions, shock, respiratory distress, dyspnea, bronchospasm, laryngeal edema, and hypotension have been reported during and within 24 hours after infusion. Immediate treatment for hypersensitivity reactions should be available during administration. Discontinue treatment immediately if signs or symptoms of anaphylaxis or severe allergic reactions occur; use caution upon rechallenge.

• Immune-mediated reactions: Severe type III immune-mediated reactions (eg, membranous glomerulonephritis) have occurred; monitor patients closely. Consider discontinuation of treatment if signs or symptoms occur; use caution with readministration (some patients have been successfully re-challenged with close supervision).

• Infusion reactions: Infusion-related reactions have been commonly reported; may be sporadic and/or severe. Serious/severe infusion reactions have included laryngeal edema, apnea, respiratory distress, dyspnea, angioedema, and anaphylactoid reaction, pyrexia, chest pain, rash, urticaria, and conjunctivitis; other reactions included chills, nausea, vomiting, pruritus, erythema, abdominal pain, hypertension, hypotension, headache, tremor, and cough. Reactions began as early as week 1 and as late as week 146 of treatment. Many patients experienced recurrent infusion reactions during multiple infusions, though not always in consecutive weeks. Patients should be premedicated with antihistamines and/or antipyretics prior to infusion; evaluate airway prior to therapy (due to possible effects of antihistamine use). Symptoms typically abated with slowing or temporary interruption of the infusion and administration of additional antihistamines, antipyretics, and, occasionally, corticosteroids. Most patients were able to complete their infusions. Subsequent infusions were managed with a slower rate of administration, treatment with additional prophylactic antihistamines, and, in the event of a more severe reaction, treatment with prophylactic corticosteroids. Discontinue treatment immediately and initiate appropriate treatment if severe reaction occurs; use caution with readministration (consider the risks/benefits of readministering following a severe reaction).

Disease-related concerns:

• Acute febrile/respiratory illness: Consider delaying treatment in patients with an acute febrile or respiratory illness.

• Patients at risk for fluid overload: Use with caution in patients who are at risk of fluid overload (patients ≤20 kg, underlying respiratory disease, or compromised cardiopulmonary function); may cause heart failure. Monitor patients closely.

• Sleep apnea: Use with caution in patients with sleep apnea (sleep apnea is common in patients with MPS VI); antihistamine pretreatment may increase the risk of apneic episodes. Evaluate airway patency prior to initiation of treatment; apnea treatment options should be readily available during the infusion and with antihistamine premedication.

• Spinal/cervical cord compression: Worsening and new-onset spinal/cervical cord compression (SCC) has been reported (SCC with resultant myelopathy is a known and anticipated serious complication of MPS VI). Monitor patients for signs and symptoms of SCC (eg, back pain, limb paralysis below the level of compression, urinary and fecal incontinence) and provide appropriate clinical care.

Special populations:

• Adults: Studies did not include patients >29 years of age.

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.

Dosage Forms: US

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

Solution, Intravenous [preservative free]:

Naglazyme: 1 mg/mL (5 mL) [contains mouse (murine) and/or hamster protein, polysorbate 80]

Generic Equivalent Available: US

No

Pricing: US

Solution (Naglazyme Intravenous)

1 mg/mL (per mL): $552.00

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, Intravenous:

Naglazyme: 1 mg/mL (5 mL) [contains mouse (murine) and/or hamster protein, polysorbate 80]

Administration: Pediatric

Parenteral: IV: Infuse via an infusion pump and PVC infusion set with in-line, low-protein-binding 0.2 micrometer filter. Pretreatment with antihistamines with or without antipyretics is recommended 30 to 60 minutes prior to infusion. Infuse over at least 4 hours; infuse 250 mL solution at 6 mL/hour for the first hour; if well-tolerated, increase rate to 80 mL/hour for the remaining 3 hours. For 100 mL infusion, use a lower rate to ensure infusion over at least 4 hours. Infusion time may be extended up to 20 hours if infusion reactions occur. If hypersensitivity or infusion-related reaction occurs, discontinue immediately.

Administration: Adult

IV: Infuse over at least 4 hours; infusions of a 250 mL total volume should begin at a rate of 6 mL/hour for the first hour, if tolerated, may increase the rate of infusion to 80 mL/hour for the remaining 3 hours. May extend infusion time up to 20 hours if infusion reactions occur. For patients requiring fluid restriction, solutions of 100 mL may be used with the rate adjusted to infuse over at least 4 hours. Administer using infusion pump and PVC (low protein-binding) infusion set with a low protein-binding 0.2 micrometer in-line filter.

Premedicate with antihistamines (with or without antipyretics) 30 to 60 minutes prior to infusion. In case of infusion-related reactions, decrease or temporarily discontinue infusion or administer additional antihistamines, antipyretics, and possibly corticosteroids. Discontinue immediately if severe reaction occurs. Patients requiring supplemental oxygen or CPAP during sleep should have these treatments readily available in case of infusion-related reaction or antihistamine-induced drowsiness.

Storage/Stability

Prior to reconstitution, store intact vials at 2°C to 8°C (36°F to 46°F). Do not freeze. Do not shake. Protect from light. Following dilution in NS, use immediately. May store under refrigeration if used within 48 hours from the time of preparation to the completion of infusion. Do not store solution for infusion at room temperature. Allow vials to reach room temperature prior to dilution. Do not keep vials at room temperature >24 hours prior to dilution. Do not heat or microwave vials.

Use

Treatment of mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) for improvement of walking and stair-climbing capacity (FDA approved in pediatric patients [age not specified] and adults ≤29 years)

Metabolism/Transport Effects

None known.

Drug Interactions

There are no known significant interactions.

Pregnancy Considerations

Information related to the use of galsulfase in pregnancy is limited (Stewart 2016).

Pregnancy may exacerbate symptoms of mucopolysaccharidosis VI, leading to adverse maternal and fetal outcomes.

Monitoring Parameters

Vital signs; presence of infusion-related reactions (chills, pruritus, urticaria, dyspnea, rash, shortness of breath, nausea, abdominal pain); in clinical studies, tests of mobility and physical function were monitored at baseline and every 6 weeks. Patients with MPS VI should be monitored for signs and symptoms of spinal/cervical cord compression (eg, back pain, paralysis of limbs below the level of compression, urinary and fecal incontinence). In patients susceptible to fluid overload (eg, weight less than 20 kg, underlying acute respiratory illness, compromised cardiac and/or respiratory function), monitor cardiac and respiratory function since congestive heart failure may occur. Evaluation of airway patency should be considered prior to start of treatment and patients using supplemental oxygen or CPAP during sleep should have these treatments available during infusion.

Mechanism of Action

Galsulfase is a recombinant form of N-acetylgalactosamine 4-sulfatase, produced in Chinese hamster cells. A deficiency of this enzyme leads to accumulation of the glycosaminoglycan dermatan sulfate in various tissues, causing progressive disease which includes decreased growth, skeletal deformities, upper airway obstruction, clouding of the cornea, heart disease, and coarse facial features. Exogenous replacement of this enzyme has been shown to improve mobility and physical function (measured by walking and stair-climbing).

Pharmacokinetics (Adult Data Unless Noted)

Note: Data based on mixed patient population of children ≥5 years and adults <29 years.

Distribution: Vz: Week 1: 103 mL/kg (range: 56 to 323 mL/kg); Week 24: 69 mL/kg (range: 59 to 2,799 mL/kg)

Half-life elimination: Week 1: Median 9 minutes (range: 6 to 21 minutes); Week 24: Median 26 minutes (range: 8 to 40 minutes)

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

  • (AR) Argentina: Naglazyme;
  • (AT) Austria: Naglazyme;
  • (AU) Australia: Naglazyme;
  • (BE) Belgium: Naglazyme;
  • (BG) Bulgaria: Naglazyme;
  • (BR) Brazil: Naglazyme;
  • (CH) Switzerland: Naglazyme;
  • (CO) Colombia: Naglazyme;
  • (CZ) Czech Republic: Naglazyme;
  • (DE) Germany: Naglazyme;
  • (EC) Ecuador: Naglazyme;
  • (EG) Egypt: Naglazyme;
  • (ES) Spain: Naglazyme;
  • (FI) Finland: Naglazyme;
  • (GB) United Kingdom: Naglazyme;
  • (HR) Croatia: Naglazyme;
  • (HU) Hungary: Naglazyme;
  • (IE) Ireland: Naglazyme;
  • (IT) Italy: Naglazyme;
  • (JP) Japan: Naglazyme;
  • (KR) Korea, Republic of: Naglazyme;
  • (LB) Lebanon: Naglazyme;
  • (LT) Lithuania: Naglazyme;
  • (MX) Mexico: Naglazyme;
  • (MY) Malaysia: Naglazyme;
  • (NL) Netherlands: Naglazyme;
  • (NO) Norway: Naglazyme;
  • (NZ) New Zealand: Naglazyme;
  • (PE) Peru: Naglazyme;
  • (PL) Poland: Naglazyme;
  • (RU) Russian Federation: Naglazim;
  • (SA) Saudi Arabia: Naglazyme;
  • (SE) Sweden: Naglazyme;
  • (SI) Slovenia: Naglazyme;
  • (SK) Slovakia: Naglazyme;
  • (TW) Taiwan: Naglazyme;
  • (UA) Ukraine: Naglazyme
  1. Alade SL, Brown RE, Paquet A Jr. Polysorbate 80 and E-Ferol toxicity. Pediatrics. 1986;77(4):593-597. [PubMed 3960626]
  2. 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]
  3. European Medicines Agency, Nalgazyme Product Information. Available at http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/000640/WC500024289.pdf. Date accessed: May 23, 2012.
  4. Giugliani R, Harmatz P, and Wraith JE, "Management Guidelines for Mucopolysaccharidosis VI," Pediatrics, 2007, 120(2):405-18. [PubMed 17671068]
  5. Giugliani R, Lampe C, Guffon N, et al. Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)--10-year follow-up of patients who previously participated in an MPS VI Survey Study. Am J Med Genet A. 2014;164A(8):1953-1964. [PubMed 24764221]
  6. Harmatz P, Giugliani R, Schwartz IV, et al, "Long-Term Follow-Up of Endurance and Safety Outcomes During Enzyme Replacement Therapy for Mucopolysaccharidosis VI: Final Results of Three Clinical Studies of Recombinant Human N-Acetylgalactosamine 4-Sulfatase," Mol Genet Metab, 2008, 94(4):469-75. [PubMed 18502162]
  7. Harmatz P, Ketteridge D, Giugliani R, et al, "Direct Comparison of Measures of Endurance, Mobility, and Joint Function During Enzyme-Replacement Therapy of Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome): Results After 48 Weeks in a Phase 2 Open-Label Clinical Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase," Pediatrics, 2005, 115(6):e681-9. [PubMed 15930196]
  8. Harmatz P, Whitley CB, Waber L, et al, "Enzyme Replacement Therapy in Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome)," J Pediatr, 2004, 144:574-80. [PubMed 15126989]
  9. Harmatz PR, Garcia P, Guffon N, et al. Galsulfase (Naglazyme®) therapy in infants with mucopolysaccharidosis VI. J Inherit Metab Dis. 2014;37(2):277-287. [PubMed 24108527]
  10. Hendriksz CJ, Giugliani R, Harmatz P, et al, "Design, Baseline Characteristics, and Early Findings of the MPS VI (Mucopolysaccharidosis VI) Clinical Surveillance Program (CSP)," J Inherit Metab Dis, 2011. [PubMed 22127392]
  11. Isaksson M, Jansson L. Contact allergy to Tween 80 in an inhalation suspension. Contact Dermatitis. 2002;47(5):312-313. [PubMed 12534540]
  12. Lucente P, Iorizzo M, Pazzaglia M. Contact sensitivity to Tween 80 in a child. Contact Dermatitis. 2000;43(3):172. [PubMed 10985636]
  13. Naglazyme (galsulfase) [prescribing information]. Novato, CA: BioMarin Pharmaceutical Inc; December 2019.
  14. Shelley WB, Talanin N, Shelley ED. Polysorbate 80 hypersensitivity. Lancet. 1995;345(8980):1312-1313. [PubMed 7746084]
  15. Stewart FJ, Bentley A, Burton BK, et al. Pregnancy in patients with mucopolysaccharidosis: a case series. Mol Genet Metab Rep. 2016;8:111-115. doi: 10.1016/j.ymgmr.2016.08.002. [PubMed 27622143]
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