Galsulfase

Mucopolysaccharide storage disorders are caused by the deficiency of specific lysosomal enzymes required for the catabolism of GAG. MPS VI is characterized by the absence or marked reduction in N-acetylgalactosamine 4-sulfatase.

• NAGLAZYME is indicated for patients with Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome). NAGLAZYME has been shown to improve walking and stair-climbing capacity.

• None. None.contraindicated

Administration of NAGLAZYME should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. ( 2.1 ) The recommended dosage is 1 mg per kg of body weight administered once weekly as an intravenous infusion. ( 2.2 ) 2.1 Recommendations Prior to NAGLAZYME Treatment Administration of NAGLAZYME should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis [see Warnings and Precautions (5.1) ] . Initiate NAGLAZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment [see Warnings and Precautions (5.1) ] . Pretreatment with antihistamines with or without antipyretics is recommended 30 to 60 minutes prior to the start of the infusion [see Warnings and Precautions (5.5) ]. 2.2 Recommended Dosage and Administration The recommended dosage regimen of NAGLAZYME is 1 mg per kg of body weight administered once weekly as an intravenous infusion. The total volume of the infusion should be delivered over a period of time of no less than 4 hours. NAGLAZYME should be diluted with 0.9% Sodium Chloride Injection, USP, to a final volume of 250 mL and delivered by controlled intravenous infusion using an infusion pump. The initial infusion rate should be 6 mL per hour for the first hour. If the infusion is well tolerated, the rate of infusion may be increased to 80 mL per hour for the remaining 3 hours.

Immune-Mediated Reactions : Immune-mediated reactions can occur with NAGLAZYME. Monitor patients for the development of immune complex-mediated reactions while receiving NAGLAZYME. ( 5.2 ) Risk of Acute Cardiorespiratory Failure : Caution should be exercised when administering NAGLAZYME to patients susceptible to fluid volume overload. Consider a decreased total infusion volume and infusion rate when administering NAGLAZYME to these patients. Appropriate medical monitoring and support measures should be available during infusion. ( 2.2 , 5.3 ) Acute Respiratory Complications : Sleep apnea is common in MPS VI patients and antihistamine pretreatment may increase the risk of apneic episodes. Appropriate respiratory support should be available during infusion. ( 5.4 ) Infusion Reactions : Pretreatment with antihistamines with or without antipyretics is recommended prior to the start of infusion to reduce the risk of infusion-reactions. If infusion reactions occur, decreasing the infusion rate, temporarily stopping the infusion, or administering additional antihistamines and/or antipyretics is recommended. ( 2.2 , 5.5 ) 5.1 Hypersensitivity Reactions Including Anaphylaxis Life-threatening hypersensitivity reactions, including anaphylaxis, have been observed in patients treated with enzyme replacement therapies, including NAGLAZYME. These reactions have occurred during and up to 24 hours after completion of the NAGLAZYME infusion.

Serious and/or clinically significant adverse reactions described elsewhere in labeling include: Hypersensitivity Reactions Including Anaphylaxis [see Warnings and Precautions ( 5.1 )] Immune-Mediated Reactions [see Warnings and Precautions ( 5.2 )] Risk of Acute Cardiorespiratory Failure [see Warnings and Precautions ( 5.2 )] Acute Respiratory Complications Associated with Administration [see Warnings and Precautions ( 5.4 )] Infusion Reactions [see Warnings and Precautions ( 5.5 )] Spinal or Cervical Cord Compression [see Warnings and Precautions ( 5.6 )] The most common adverse reactions (≥10%) are: rash, pain, urticaria, pyrexia, pruritus, chills, headache, nausea, vomiting, abdominal pain and dyspnea. The most common adverse reactions requiring interventions are infusion-related reactions. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact: BioMarin Pharmaceutical Inc. at 1-866-906-6100 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 observed in the clinical trials of another drug and may not reflect the rates observed in clinical practice. NAGLAZYME was studied in a randomized, double-blind, placebo-controlled trial in which 19 patients received weekly infusions of 1 mg/kg NAGLAZYME and 20 patients received placebo; of the 39 patients 66% were female, and 62% were White, non-Hispanic. Patients were aged 5 years to 29 years.

8.1 Pregnancy Risk Summary Available data from a pregnancy sub-study within the MPS VI Clinical Surveillance Program and case reports with NAGLAZYME use in pregnant women have not identified a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. In animal reproduction studies, galsulfase administered intravenously to pregnant rats and rabbits during the period of organogenesis, showed no evidence of harm to the fetus at doses of about 0.5 and 0.97 times, respectively for rats and rabbits, the recommended human dose of 1 mg/kg based on body surface area (see Data) . The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defects, loss, or other adverse outcomes. 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 embryo/fetal risk Pregnancy can exacerbate preexisting clinical manifestations of MPS and lead to adverse pregnancy outcomes for both mother and fetus.

Metabolism

The pharmacokinetic parameters of galsulfase were evaluated in 13 patients with MPS VI who received 1 mg/kg of NAGLAZYME as a weekly 4-hour infusion for 24 weeks. The pharmacokinetic parameters at Week 1 and Week 24 are shown in Table 2.

• May 31, 2005BLABLA125117Biomarin

1. 1Pyrexia40811%
2. 2Pneumonia2987.9%
3. 3Cough2225.9%
4. 4Dyspnoea1905.0%
5. 5Spinal Cord Compression1594.2%
6. 6Vomiting1303.4%
7. 7Oxygen Saturation Decreased1243.3%
8. 8Fatigue1072.8%
9. 9Headache932.5%
10. 10Influenza912.4%
11. 11Death882.3%
12. 12Pain882.3%
13. 13Nasopharyngitis862.3%
14. 14Fall792.1%
15. 15Rhinorrhoea762.0%

The 3 most recently updated of 3 ClinicalTrials.gov registrations naming Galsulfase as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
  Recruiting · Phase 1 · Interventional · 10 enrolled · University of California, San Francisco
  NCT04532047updated 2026-03-17
• Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
  Terminated · Interventional · 1 enrolled · Masonic Cancer Center, University of Minnesota
  NCT02156674updated 2020-12-23
• A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
  Completed · Phase 4 · Interventional · 4 enrolled · BioMarin Pharmaceutical
  NCT00299000updated 2011-07-22

Frequently asked questions

How does Galsulfase work?

Mucopolysaccharide storage disorders are caused by the deficiency of specific lysosomal enzymes required for the catabolism of GAG. MPS VI is characterized by the absence or marked reduction in N-acetylgalactosamine 4-sulfatase.

What is Galsulfase used for?

According to FDA labeling, Galsulfase carries indications including: NAGLAZYME is indicated for patients with Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome). NAGLAZYME has been shown to improve walking and stair-climbing capacity.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Galsulfase?

Galsulfase is classified as Enzymes, Hydrolytic Lysosomal Glycosaminoglycan-specific Enzyme, Increased Lysosomal Function.

What are the brand names for Galsulfase?

Galsulfase is marketed under brand names including Naglazyme.

What are the contraindications for Galsulfase?

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