WARNING: SEVERE HYPERSENSITIVITY REACTIONS, INFUSION-ASSOCIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS
HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
Patients treated with POMBILITI have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available during POMBILITI administration. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, POMBILITI should be discontinued immediately, and appropriate medical treatment should be initiated. In patients with severe hypersensitivity reaction, desensitization measures to POMBILITI may be considered. The risks and benefits of readministering POMBILITI following severe hypersensitivity reaction should be considered. If mild or moderate hypersensitivity reaction occurs, the infusion rate may be slowed or temporarily stopped. Prior to POMBILITI administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids.
INFUSION-ASSOCIATED REACTIONS (IARs)
Patients treated with POMBILITI have experienced severe IARs. If severe IARs occur, immediately discontinue the POMBILITI infusion, initiate appropriate medical treatment, and assess the benefits and risks of readministering POMBILITI following severe IARs. If mild or moderate IARs occur regardless of pretreatment, decreasing the infusion rate or temporarily stopping the infusion may ameliorate the symptoms. IARs may still occur in patients after receiving pretreatment.
Patients with an acute underlying illness at the time of POMBILITI infusion may be at greater risk for IARs. Patients with advanced Pompe disease may have compromised cardiac and respiratory function, which may predispose them to a higher risk of severe complications from IARs.
RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS
Patients susceptible to fluid volume overload, or those with acute underlying respiratory illness or compromised cardiac or respiratory function for whom fluid restriction is indicated may be at risk of serious exacerbation of their cardiac or respiratory status during POMBILITI infusion. More frequent monitoring of vitals should be performed during POMBILITI infusion in such patients.
CONTRAINDICATION
POMBILITI in combination with OPFOLDA is contraindicated in pregnancy.
EMBRYO-FETAL TOXICITY
Based on findings from animal reproduction studies, POMBILITI in combination with OPFOLDA may cause embryo-fetal harm when administered to a pregnant female and is contraindicated during pregnancy. Verify the pregnancy status in females of reproductive potential prior to initiating treatment with POMBILITI in combination with OPFOLDA. Advise females of reproductive potential to use effective contraception during treatment with POMBILITI in combination with OPFOLDA and for at least 60 days after the last dose.
RISKS ASSOCIATED WITH POMBILITI AND OPFOLDA
POMBILITI and OPFOLDA must be administered in combination.
ADVERSE REACTIONS
The most common adverse reactions (≥5%) reported in the pooled safety population of patients treated with POMBILITI in combination with OPFOLDA in the 3 clinical trials were headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia.
To report SUSPECTED ADVERSE REACTIONS, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
LACTATION
Advise females that breastfeeding is not recommended while on treatment with POMBILITI in combination with OPFOLDA.
INDICATION
POMBILITI® (cipaglucosidase alfa-atga) in combination with OPFOLDA® (miglustat) is indicated for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).
Please see full Prescribing Information, including BOXED WARNING, for POMBILITI and full Prescribing Information for OPFOLDA.
