POMBILITI (cipaglucosidase alfa-atga)

There are 1 contraindications. Absolute contraindication.

Contraindication List
Lactation

There are 3 severe contraindications. Adequate patient monitoring is recommended for safer drug use.

Severe List
Acute respiratory failure
Chronic heart failure
Pregnancy

There are 0 moderate contraindications.

There are 6 severe adverse reactions.

More Frequent	Less Frequent
None.	Anaphylaxis Hypersensitivity drug reaction Paradoxical bronchospasm Pharyngeal edema Tongue swelling

Rare/Very Rare
Hypotension

There are 30 less severe adverse reactions.

More Frequent	Less Frequent
Acute abdominal pain Diarrhea Fatigue Fever Headache disorder Nausea	Chills Dizziness Dysgeusia Dyspnea Flushing Muscle spasm Pruritus of skin Skin rash Tachycardia Urticaria

Rare/Very Rare
Arthralgia Constipation Drowsy Dyspepsia Flank pain General weakness Hypertension Malaise Myalgia Pain Paresthesia Thrombocytopenic disorder Tremor Vomiting

There is insufficient evidence with cipaglucosidase alfa-atga in combination with miglustat in pregnant women to evaluate a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. Based on findings from animal reproduction studies, cipaglucosidase alfa-atga in combination with miglustat may cause embryo-fetal harm when administered to a pregnant female and is contraindicated during pregnancy. In a rabbit embryo-fetal development study, great vessel and cardiac malformations were increased in offspring of pregnant rabbits treated with cipaglucosidase alfa-atga in combination with miglustat at 16-fold and 3-fold, respectively, of the maximum recommended human dose (MRHD) based on plasma AUC exposure.

A No Observed Adverse Effect Level (NOAEL) was not identified for the combination. In a pre- and post-natal development study in rats, increases in pup mortality were seen following maternal treatment with cipaglucosidase alfa-atga (400 mg/kg) in combination with miglustat, or with cipaglucosidase alfa-atga (400 mg/kg) alone. The NOAEL for cipaglucosidase alfa-atga alone is 150 mg/kg (5-fold the cipaglucosidase alfa-atga MRHD margin). A NOAEL for the combination of cipaglucosidase alfa-atga and miglustat was not identified.

There are no data on the presence of cipaglucosidase alfa-atga (alone or in combination with miglustat) in human milk, the effects on the breast-fed child, or the effects on milk production. Cipaglucosidase alfa-atga is present in animal milk. Based on findings in animal studies, the use of cipaglucosidase alfa-atga in combination with miglustat may lead to serious adverse reactions in breast-fed infants. Advise females that breastfeeding is not recommended while receiving treatment with cipaglucosidase alfa-atga in combination with miglustat.

Clinical studies with cipaglucosidase alfa-atga did not include sufficient numbers of subjects 65 years of age and older to determine whether they respond differently than younger subjects. Of the total number of patients treated with cipaglucosidase alfa-atga in combination with miglustat in clinical trials for late-onset Pompe Disease, 17 (11%) were 65 to 74 years of age, and none were >=75 year of age.