Usual Adult Dose for Hypophosphatasia

2 mg/kg subcutaneously 3 times per week OR 1 mg/kg subcutaneously 6 times per week
Maximum dose: 9 mg/kg per week

Comments:

• For patients with perinatal/infantile-onset hypophosphatasia (HPP), an increase to 3 mg/kg three times per week may be necessary for insufficient efficacy (e.g., no improvement in respiratory status, growth, or radiographic findings).
• Injection site reactions may limit the tolerability of the 6 times per week dosing regimens.

Usual Pediatric Dose for Hypophosphatasia

2 mg/kg subcutaneously 3 times per week OR 1 mg/kg subcutaneously 6 times per week
Maximum dose: 9 mg/kg per week

Comments:

• For patients with perinatal/infantile-onset hypophosphatasia (HPP), an increase to 3 mg/kg three times per week may be necessary for insufficient efficacy (e.g., no improvement in respiratory status, growth, or radiographic findings).
• For patients weighing less than 40 kg: Do not use the 80 mg/0.8 mL vial because the systemic exposure is lower than that achieved with the lower strength vials and may not be adequate for these patients.
• Injection site reactions may limit the tolerability of the 6 times per week dosing regimens.

Renal Dose Adjustments

Data not available

Liver Dose Adjustments

Data not available

Precautions

CONTRAINDICATIONS:

• None

Dialysis

Data not available

Other Comments

Administration advice:

• Administer subcutaneously into the abdominal area, thigh, or deltoid
• Always use a new syringe and needle; do not share syringes or needles
• Rotate injection sites to reduce risk of lipodystrophy; do not administer into reddened, inflamed, or swollen areas

• Store in the refrigerator [2C to 8C (36F to 46F)] in the original carton until time of use; protect from light
• Once removed from refrigerator, use within 1 hour
• Do not freeze or shake vial; single-use only

• Administer dose within 1 hour of removing vial from the refrigerator.
• Do not use 80 mg/0.8 mL vial in patients weighing less than 40 kg.
• For injection volumes greater than 1 mL, split dose equally between 2 injections and use 2 separate injection sites.
• Vial should be clear, slightly opalescent or opalescent, colorless to slightly yellow aqueous solution; few small translucent or white particles may be present; discard vials not consistent with this description.
• Manufacturer product labeling should be consulted for weight-based dosing tables and more specific administration guidance.

• Hypophosphatasia (HPP) Registry: A voluntary registry has been established to better understand the HPP population and to monitor and evaluate the long-term treatment effects of this drug; information can be found at www.hppregistry.com.
• In clinical trials, anti-drug antibodies were detected; no correlation between anti-drug antibody titer and percent inhibition was observed. The formation of anti-drug antibody resulted in a reduction in systemic exposure.

• Signs and symptoms of hypersensitivity reactions
• Signs and symptoms of lipodystrophy
• Ophthalmologic exams and renal ultrasounds at baseline and periodically during treatment to monitor for ectopic calcifications

• Patients or caregivers should be instructed on preparation, storage, and administration of this drug; proper injection technique and site rotation are necessary to avoid injection site reactions.
• Patients or caregivers should be instructed on proper disposal of needles and syringes; with each dose, a new syringe and needle should be used and needles and syringes should never be shared.
• If signs or symptoms of an allergic reaction occur, patients should seek immediate medical advice.
• Tell your health care provider if you notice skin thickening or pits at injection sites.