Vimizim (elosulfase alfa)
Metabolic Disease Enzyme Replacement - Mucopolysaccharidosis Intravenous Vial (Ml)
Last reviewed · Source: First Databank (FDB) drug data
Overview
Elosulfase alfa is a biosynthetic (recombinant DNA origin) form of human N-acetylgalactosamine-6-sulfatase (GALNS), a lysosomal enzyme that catalyzes the hydrolysis of the glycosaminoglycans keratan sulfate and chondroitin-6-sulfate.
Elosulfase alfa is a biosynthetic (recombinant DNA origin) form of human N-acetylgalactosamine-6-sulfatase, a lysosomal enzyme that catalyzes the hydrolysis of sulfate from either galactose-6-sulfate or N-acetyl-galactosamine-6-sulfate on the nonreducing ends of the glycosaminoglycans keratan sulfate and chondroitin-6-sulfate. The drug is produced using a Chinese hamster ovary cell line.
Mucopolysaccharidosis IVA (MPS IVA, Morquio A syndrome) is a lysosomal storage disorder characterized by a deficiency of the N-acetylgalactosamine-6-sulfatase (GALNS) enzyme. (See Uses: Mucopolysaccharidosis IVA.) In patients with this condition, elosulfase alfa provides an exogenous source of GALNS for catabolism of accumulated glycosaminoglycans (keratan sulfate and chondroitin-6-sulfate). Cellular uptake of elosulfase alfa into lysosomes is mediated by the binding of mannose-6-phosphate-terminated oligosaccharide chains of the drug to mannose-6-phosphate receptors. In human primary chondrocytes from MPS IVA patients, treatment with elosulfase alfa induced clearance of keratan sulfate lysosomal storage from the chondrocytes.
Elosulfase alfa appears to exhibit time-dependent pharmacokinetics. In a study that evaluated pharmacokinetic parameters after the initial infusion (week 0) and after the infusion at 22 weeks in patients receiving elosulfase alfa 2 mg/kg once weekly, systemic exposure, peak plasma concentrations, time to peak plasma concentrations, and half-life of the drug increased over time. Mean area under the plasma concentration-time curve (AUC) and peak plasma concentrations increased by 2.8- and 2.9-fold, respectively, at week 22 compared with week 0.
Mean elimination half-life increased from approximately 8 to 36 minutes, and time to peak plasma concentrations increased from 172 to 202 minutes, from week 0 to week 22, respectively. These time-dependent pharmacokinetic changes are likely related to the formation of neutralizing antibodies. Although elosulfase alfa has a short half-life, the intracellular half-life of the drug in human Morquio fibroblasts is estimated to be 5-7 days.
USES: Elosulfase alfa is used to treat a certain inherited condition (mucopolysaccharidosis type IVA-Morquio A syndrome) that causes a buildup of certain substances (glycosaminoglycans-GAGs) in the body. Elosulfase alfa works by breaking down GAGs in the body.
Generic name: elosulfase alfa
Pronunciation: (EL-oh-SUL-fase AL-fa)
Drug class: Metabolic Disease Enzyme Replacement - Mucopolysaccharidosis
Therapeutic class: Metabolic Disease Enzyme Replacement Agents
Reference product & biosimilars
No reference, biosimilar or interchangeable associated with this drug.
Today’s intelligence on Vimizim
Indications
The following indications are listed for Vimizim:
- Mucopolysaccharidosis Type Iv-A
Also known as: Classic Morquio Syndrome, Galactosamine-6-Sulfatase Deficiency, Galactose-6-Sulfatase Deficiency, Morquio A Mucopolysaccharidoses, Mucopolysaccharidosis, Type Iva, N-Acetylgalactosamine-6-Sulfatase Deficiency.
Dosing
Dosage of elosulfase alfa is expressed in mg. The specific activity of elosulfase alfa is 2.6-6 units/mg, with 1 unit defined as the amount of enzyme that results in the conversion of 1 mcmol of sulfated monosaccharide substrate D-galactopyranoside-6-sulfate to desulfated-galactose and free sulfate per minute at 37degreesC.
The recommended dosage of elosulfase alfa for the treatment of mucopolysaccharidosis IVA (MPS IVA) in adults and children 5 years of age and older is 2 mg/kg given by IV infusion once weekly.
Elosulfase alfa is administered by IV infusion.
Elosulfase alfa should be administered using a low-protein-binding infusion set equipped with a low-protein-binding 0.2-mcm inline filter. Compatibility with other drugs has not been evaluated; therefore, elosulfase alfa must not be infused through the same IV tubing with other drugs.
| Drug label | Dosing type | Instructions |
|---|---|---|
| Vimizim 5 Mg/5 Ml Vial | Maintenance | Infuse 2 mg/kg over at least 4.5 hour(s) by intravenous route once weekly. |
No dosing information is available for this tab.
Drug interactions
Formal drug interaction studies have not been performed to date.
There are no contraindication interactions for this drug.
There are no severe interactions for this drug.
There are no moderate interactions for this drug.
Contraindications
The manufacturer states that there are no known contraindications to the use of elosulfase alfa.
There are no contraindication contraindications for this drug.
There are no severe contraindications for this drug.
- No disease contraindications
Adverse reactions
Adverse effects reported in more than 10% of patients receiving elosulfase alfa 2 mg/kg once weekly in clinical studies include pyrexia, vomiting, headache, nausea, abdominal pain, chills, and fatigue.
Less Frequent
- Anaphylaxis
Rare or Very Rare
- Hypersensitivity drug reaction
More Frequent
- Acute abdominal pain
- Fever
- Headache disorder
- Nausea
- Vomiting
Less Frequent
- Chills
- Fatigue
Precautions
No enhanced messaging available.
The majority of patients included in the principal efficacy study of elosulfase alfa were pediatric patients ranging in age from 5-11 years (53%) and 12-17 years (27%). Safety and efficacy of the drug have not been established in pediatric patients younger than 5 years of age.
Category C. (See Users Guide)
There are no adequate and well-controlled studies of elosulfase alfa in pregnant women; the drug should be used during pregnancy only if the potential benefits justify the potential risks to the fetus. Animal reproductive studies have not demonstrated any evidence of adverse embryofetal effects; however, maternal toxicity, increased pup mortality, and increased stillbirths have been observed.
The Morquio A Registry collects data on pregnant women with MPS IVA who are treated with elosulfase alfa. For additional information or to enroll in the registry, patients may call 800-983-4587 or contact MARS@bmrn.com.
Elosulfase alfa is distributed into milk in rats. It is not known whether the drug is distributed into human milk. The known benefits of breastfeeding should be considered along with the mother's clinical need for elosulfase alfa and any potential adverse effects of the drug or disease on the infant. Caution is advised when elosulfase alfa is used in nursing women.
The Morquio A Registry collects data on nursing women with MPS IVA who are treated with elosulfase alfa. For additional information or to enroll in the registry, patients may call 800-983-4587 or contact MARS@bmrn.com.
Clinical studies of elosulfase alfa did not include patients 65 years of age or older. It is not known whether geriatric patients respond differently to the drug than younger patients.
ICD codes
| Code | Type | Description |
|---|---|---|
| Mucopolysaccharidosis type Iv-A | ||
| E76.210 | ICD10CM | Morquio A mucopolysaccharidoses |
ICD code information from First Databank (FDB).
Formulary status
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Last reviewed · Source: First Databank (FDB) drug data
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