Mucopolysaccharidosis VII

The recommended dose of vestronidase alfa is 4 mg/kg of body weight administered by intravenous infusion every two weeks.

To minimise the risk of hypersensitivity reactions, a non-sedating antihistamine with or without an antipyretic medicinal product should be administered 30-60 minutes prior to the start of the infusion (see section 4.4). Infusion should be avoided if the patient has an acute febrile or respiratory illness at the time.

The total diluted volume of the solution for infusion should be administered with a rate titration regimen over approximately 4 hours.

The rate of infusion should be as follows: in the first hour, 2.5% of the total volume will be infused, with the balance infused over the subsequent three hours. Any dead space in the lines should be accounted for to ensure 2.5% of the total infusion volume is delivered into the patient’s bloodstream during the first hour of infusion. The lowest rate administered to a patient in the clinical development program was 0.5 mL/hour during the first 30 minutes of infusion, followed by 1 mL/hour over the next 30 minutes, equalling 0.75 mL as the lowest total volume infused during the first hour.

Do not flush the line containing vestronidase alfa to avoid a rapid bolus of infused enzyme. Due to the low infusion rate, additional sodium chloride 9 mg/mL (0.9%) solution for infusion may be added through a separate line (piggyback or Y tube) to maintain sufficient intravenous flow. After the first hour, the rate can be increased to infuse the remainder of the solution for infusion over 3 hours as tolerated according to the recommended rate guidelines.