VIMIZIM

What it is

Vimizim contains elosulfase alfa (rch), a concentrated solution for injection at 1 mg/mL . Elosulfase alfa is a purified human enzyme produced by recombinant DNA technology in a Chinese hamster ovary cell line . It is a lysosomal enzyme that hydrolyses sulfate from either galactose-6-sulfate or N-acetyl-galactosamine-6-sulfate on the non-reducing ends of the glycosaminoglycans keratan sulfate and chondroitin sulfate .

Approved indications

— Treatment of mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome)

Dosing overview

The recommended dosage for Vimizim is 2 mg/kg of body weight administered once a week . The total volume of the infusion should be delivered over approximately 4 hours . Pre-treatment with antihistamines with or without antipyretics is recommended 30–60 minutes prior to start of infusion . Vimizim must be diluted with sodium chloride 9 mg/mL (0.9%) solution for injection to a final volume of 100 mL or 250 mL based on the patient's weight, with patients weighing less than 25 kg receiving 100 mL and patients weighing 25 kg or more receiving 250 mL .

Key safety warnings

Anaphylaxis and severe allergic reactions have been reported in clinical studies, and appropriate medical support must be readily available when Vimizim is administered . In clinical trials, anaphylaxis was reported as early as 30 minutes from the start of infusion and up to three hours after infusion, and anaphylaxis occurred as late into treatment as the 47th infusion . In clinical trials, 96% of patients treated with Vimizim experienced infusion reactions . In patients who experienced infusion reactions, subsequent infusions were managed with slower infusion rates, treatment with additional prophylactic antihistamines and, in the event of a more severe reaction, treatment with prophylactic corticosteroids . Spinal/cervical cord compression is a known and serious complication of MPS IVA and may occur as part of the natural history of the disease . Patients with MPS IVA should be monitored for signs and symptoms of spinal/cervical cord compression (including back pain, paralysis of limbs below the level of compression, urinary and fecal incontinence) and given appropriate clinical care . Patients with acute febrile or respiratory illness at the time of Vimizim infusion may be at higher risk of life-threatening complications from hypersensitivity reactions, and careful consideration should be given to the patient's clinical status prior to administration . Sleep apnoea is common in MPS IVA patients, and evaluation of airway patency should be considered prior to initiation of treatment with Vimizim .

Contraindications

Severe or life-threatening hypersensitivity to the active substance or to any of the excipients is a contraindication, if hypersensitivity is not controllable .

Regulatory history

Vimizim (elosulfase alfa 1 mg/mL concentrated solution for injection) was first listed on the ARTG on 9 December 2014 .