Animal studies have revealed evidence of embryofetal effects due to maternal toxicity. IV administration in pregnant mice resulted in an immunologic response (including an anaphylactoid response) in some dams at 17 times the human steady-state AUC at the biweekly dose of 20 mg/kg for late-onset Pompe disease (LOPD) patients weighing at least 30 kg (or 10 times the human steady-state AUC at the biweekly dose of 40 mg/kg for LOPD patients weighing less than 30 kg); increased postimplantation loss and mean number of late resorptions were observed. This drug was not transferred through the placenta from maternal to fetal circulation in mice, suggesting embryofetal effects were due to maternal toxicity relating to the immunologic response; the maternal no observed adverse effect level (NOAEL) was 17 times the human AUC and the developmental NOAEL was 4.8 times the human AUC. IV administration in pregnant rabbits resulted in no adverse effects in fetuses at 91 times the human steady-state AUC at the biweekly dose of 20 mg/kg for LOPD patients weighing at least 30 kg (or 50 times the human steady-state AUC at the biweekly dose of 40 mg/kg for LOPD patients weighing less than 30 kg). There are no controlled data in human pregnancy.

Pregnant women exposed to this drug (or their health care providers) should report this exposure by calling 1-800-745-4447, extension 15500.

AU TGA pregnancy category B1: Drugs which have been taken by only a limited number of pregnant women and women of childbearing age, without an increase in the frequency of malformation or other direct or indirect harmful effects on the human fetus having been observed. Studies in animals have not shown evidence of an increased occurrence of fetal damage.

US FDA pregnancy category Not Assigned: The US FDA has amended the pregnancy labeling rule for prescription drug products to require labeling that includes a summary of risk, a discussion of the data supporting that summary, and relevant information to help health care providers make prescribing decisions and counsel women about the use of drugs during pregnancy. Pregnancy categories A, B, C, D, and X are being phased out.

This drug should be used during pregnancy only if the benefits to the mother outweigh the risks, including those to the fetus.

AU TGA pregnancy category: B1
US FDA pregnancy category: Not assigned.

Risk summary: Insufficient data available on use of this drug in pregnant women to inform a drug-related risk; however, postmarketing reports and published case reports on alglucosidase alfa (another hydrolytic lysosomal glycogen-specific enzyme replacement therapy) have not identified a drug-related risk of adverse pregnancy outcomes.

Comments:
-The continuation of therapy for Pompe disease during pregnancy should be individualized to the patient.
-Untreated Pompe disease has been associated with worsening respiratory and musculoskeletal symptoms in some pregnant women.

See references

An alternate drug may be preferred, particularly while breastfeeding newborn or preterm infants.
-Safety has not been established; this drug should be used during breastfeeding only if the benefits to the mother outweigh the risks, including those to the breastfed child.

Excreted into human milk: Unknown
Excreted into animal milk: Unknown

Comments:
-No information is available on the clinical use of this drug during breastfeeding.
-Developmental and health benefits of breastfeeding should be considered as well as the mother's clinical need for this drug.
-The effects in the nursing infant are unknown; potential side effects in the breastfed child due to this drug or the mother's underlying condition should be considered.
-Lactating women exposed to this drug (or their health care providers) should report this exposure by calling 1-800-745-4447, extension 15500.

Since this drug is a large protein molecule (molecular weight about 124 kilodaltons), the amount in milk is likely to be very low; absorption is unlikely because it is probably partially destroyed in the infant's gastrointestinal tract and poorly absorbed orally.

Available published literature suggests alglucosidase alfa (another hydrolytic lysosomal glycogen-specific enzyme replacement therapy) is present in human milk.

See references