Elosulfase Alfa

Mucopolysaccharidoses comprise a group of lysosomal storage disorders caused by the deficiency of specific lysosomal enzymes required for the catabolism of glycosaminoglycans (GAG). Mucopolysaccharidosis IVA (MPS IVA, Morquio A Syndrome) is characterized by the absence or marked reduction in N -acetylgalactosamine-6-sulfatase activity.

• VIMIZIM (elosulfase alfa) is indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome). VIMIZIM is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).

• None. None.contraindicated

Administration of VIMIZIM should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. ( 2.1 ) 2 mg per kg body weight administered once every week as an intravenous infusion over a minimum of 3.5 to 4.5 hours, based on infusion volume. ( 2.2 , 2.4 ) See the full prescribing information for administration modifications due to hypersensitivity reactions. ( 2.3 ) 2.1 Important Administration Instructions Administration of VIMIZIM should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis [see Warnings and Precautions (5.1) ] . Initiate VIMIZIM in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation [see Warnings and Precautions (5.1) ] . This product must be diluted prior to administration and administered using a low-protein binding infusion set equipped with a low-protein binding 0.2 micrometer (µm) in-line filter. Consider pre-medicating with antihistamines, with or without antipyretics, 30 to 60 minutes prior to the start of the infusion [see Warnings and Precautions (5.1) ] . 2.2 Recommended Dosage The recommended dosage of VIMIZIM is 2 mg/kg administered intravenously over a minimum range of 3.5 to 4.5 hours (based on infusion volume) once every week.

Risk of Acute Respiratory Complications: Patients with acute febrile or respiratory illness may be at higher risk of life-threatening complications from hypersensitivity reactions. Careful consideration should be given to the patient's clinical status prior to administration of VIMIZIM and consider delaying the VIMIZIM infusion. ( 5.2 ) 5.1 Hypersensitivity Reactions Including Anaphylaxis Life-threatening hypersensitivity reactions, including anaphylaxis, have been reported in patients treated with enzyme replacement therapies, including VIMIZIM. In premarketing clinical trials, 18 of 235 (7.7%) patients treated with VIMIZIM experienced signs and symptoms consistent with anaphylaxis. These 18 patients experienced 26 anaphylactic reactions during infusion with signs and symptoms including cough, erythema, throat tightness, urticaria, flushing, cyanosis, hypotension, rash, dyspnea, chest discomfort, and gastrointestinal symptoms (e.g., nausea, abdominal pain, retching, and vomiting) in conjunction with urticaria. These cases of anaphylaxis occurred as early as 30 minutes from the start of infusion and up to three hours after infusion. Anaphylaxis occurred as late into treatment as the 47 th infusion. In clinical trials with VIMIZIM, 44 of 235 (18.7%) patients experienced hypersensitivity reactions, including anaphylaxis. Hypersensitivity reactions have occurred as early as 30 minutes from the start of infusion but as late as six days after infusion.

The following serious adverse reactions are described below and elsewhere in the labeling: Hypersensitivity Reactions Including Anaphylaxis [see Warnings and Precautions (5.1) ] . Risk of Acute Respiratory Complications [see Warnings and Precautions (5.2) ] . Spinal or Cervical Cord Compression [see Warnings and Precautions (5.3) ] . Most common adverse reactions (≥10%) are: pyrexia, vomiting, headache, nausea, abdominal pain, chills, and fatigue. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact BioMarin 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 in the clinical trials of another drug and may not reflect the rates observed in practice. A 24-week, randomized, double-blind, placebo-controlled clinical trial of VIMIZIM was conducted in 176 patients with MPS IVA, ages 5 to 57 years old. Approximately half of the patients (49%) were male. Of the 176 patients, 65% were White, 23% Asian, 3% Black, and 10% Other race. The majority of patients (78%) were non-Hispanic. Patients were randomized to three treatment groups: VIMIZIM 2 mg/kg once per week (n=58), VIMIZIM 2 mg/kg once every other week (n=59), or placebo (n=59). All patients were treated with antihistamines prior to each infusion.

8.1 Pregnancy Risk Summary Available data from published case reports, a registry with a pregnancy sub-study and pharmacovigilance reports with VIMIZIM use during pregnancy have not identified a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. Limitations of the available data include a small number of exposed cases and missing data. In animal reproduction studies, no effects on embryo-fetal development were observed in rats given daily administration of elosulfase alfa up to 33 times the human steady-state AUC (area under the concentration-time curve) at the recommended human weekly dose pre-mating and through the period of organogenesis. No effects on embryo-fetal development were observed in rabbits given daily administration of elosulfase alfa at doses up to 8 times the human steady-state AUC at the recommended weekly dose during organogenesis, which produced maternal toxicity. A dose-dependent increase in stillbirths was observed when elosulfase alfa was administered daily in rats during organogenesis through lactation at doses 5 times the human steady-state AUC at the recommended human weekly dose. An increase in pup mortality was observed at doses producing maternal toxicity. The background risk of major birth defects and miscarriage for the indicated population is unknown.

Metabolism

The pharmacokinetics of elosulfase alfa were evaluated in 23 patients with MPS IVA who received intravenous infusions of VIMIZIM 2 mg/kg once weekly, over approximately 4 hours, for 22 weeks. Eleven patients were aged 5 to 11 years, six were aged 12 to 17 years, and six were aged 18 to 41 years.

• Feb 14, 2014BLABLA125460Biomarin Pharm

1. 1Pyrexia71914%
2. 2Cough3396.4%
3. 3Vomiting3256.2%
4. 4Malaise2855.4%
5. 5Headache2544.8%
6. 6Dyspnoea2284.3%
7. 7Nausea2224.2%
8. 8Pain2224.2%
9. 9Nasopharyngitis1883.6%
10. 10Fatigue1863.5%
11. 11Pneumonia1613.1%
12. 12Product Dose Omission1552.9%
13. 13Oxygen Saturation Decreased1502.8%
14. 14Pain In Extremity1352.6%
15. 15Oropharyngeal Pain1342.5%

The 7 most recently updated of 7 ClinicalTrials.gov registrations naming Elosulfase Alfa 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
• Natural History of Atypical Morquio A Disease
  Completed · Observational · 7 enrolled · GOIZET
  NCT03204370updated 2025-10-03
• A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
  Completed · Observational · 418 enrolled · BioMarin Pharmaceutical
  NCT02294877updated 2024-03-05
• BMN 110 Phase 3B in Australian Patients
  Completed · Phase 3 · Interventional · 13 enrolled · BioMarin Pharmaceutical
  NCT01966029updated 2019-09-26
• Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
  Terminated · Phase 2 · Interventional · 25 enrolled · BioMarin Pharmaceutical
  NCT01609062updated 2016-02-01
• Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
  Terminated · Phase 2 · Interventional · 16 enrolled · BioMarin Pharmaceutical
  NCT01697319updated 2016-01-12
• A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
  Completed · Phase 3 · Interventional · 177 enrolled · BioMarin Pharmaceutical
  NCT01275066updated 2014-07-07

Frequently asked questions

How does Elosulfase Alfa work?

Mucopolysaccharidoses comprise a group of lysosomal storage disorders caused by the deficiency of specific lysosomal enzymes required for the catabolism of glycosaminoglycans (GAG). Mucopolysaccharidosis IVA (MPS IVA, Morquio A Syndrome) is characterized by the absence or marked reduction in N -acetylgalactosamine-6-sulfatase activity.

What is Elosulfase Alfa used for?

According to FDA labeling, Elosulfase Alfa carries indications including: VIMIZIM (elosulfase alfa) is indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome). VIMIZIM is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Elosulfase Alfa?

Elosulfase Alfa is classified as Enzymes, Hydrolytic Lysosomal Glycosaminoglycan-specific Enzyme, Glycosaminoglycan Metabolism Increase, Increased Lysosomal Function.

What are the brand names for Elosulfase Alfa?

Elosulfase Alfa is marketed under brand names including Vimizim.

What are the contraindications for Elosulfase Alfa?

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