Cipaglucosidase Alfa

Cipaglucosidase Alfa is a prescription medication indicated in combination with miglustat (Opfolda) for adults with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) who weigh more than 40 kg and are not improving on their current enzyme replacement therapy (ERT).

• Cipaglucosidase Alfa is available under the following different brand names: Pombiliti, cipaglucosidase Alfa-atga.

Common side effects of Cipaglucosidase Alfa include:

• headache 
• diarrhea
• fatigue
• nausea
• abdominal pain
• pyrexia

Serious side effects of Cipaglucosidase Alfa include:

• difficulty breathing 
• rash
• swelling of legs or arms 
• swelling or feeling of tightness in the throat 
• tongue swelling
• itching
• flushing or chills

Rare side effects of Cipaglucosidase Alfa include:

• none 

Seek medical care or call 911 at once if you have the following serious side effects:

• Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, coordination loss, unsteady, stiff muscles, high fever, profuse sweating, or tremors.
• Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights.
• Serious heart symptoms include fast, irregular, or pounding heartbeats; fluttering in the chest; shortness of breath; sudden dizziness, lightheadedness, or passing out.

This is not a complete list of side effects and other serious side effects or health problems that may occur because of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.

Adult dosage

Injection, lyophilized powder for reconstitution

• 105 mg/single-dose vial

Pompe disease

Adult dosage

• Dose based on actual body weight
• 20 mg/kg IV every other week
• Start miglustat combination with Cipaglucosidase Alfa 2 weeks after the last ERT dose
• Take miglustat 1 hour before administering Cipaglucosidase Alfa IV

Dosage Considerations – Should be Given as Follows: 

• See “Dosages”

If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider, or pharmacist first.

• Cipaglucosidase Alfa has no noted severe interactions with any other drugs
• Cipaglucosidase Alfa has no noted serious interactions with any other drugs
• Cipaglucosidase Alfa has no noted moderate interactions with any other drugs
• Cipaglucosidase Alfa has no noted minor interactions with any other drugs

This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all the products you use. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your healthcare professional or doctor for additional medical advice, health questions, or concerns.

Contraindications

• Pregnancy

Effects of drug abuse

• None

Short-Term Effects

• See “What Are Side Effects Associated with Using Cipaglucosidase Alfa?”

Long-Term Effects

• See “What Are Side Effects Associated with Using Cipaglucosidase Alfa?”

Cautions

• Hypersensitivity reactions
  • Hypersensitivity reactions, including anaphylaxis, reported; ensure appropriate medical support measures are readily available, including cardiopulmonary resuscitation equipment
  • Anaphylaxis signs and symptoms included dyspnea, rash, hypotension, bronchospasm, edema, pharyngeal edema, and tongue swelling
  • Symptoms of severe hypersensitivity reactions included urticaria, pruritus, and flushing
  • See dosage and administration modifications for recommendations to discontinue, slow rate, or need premedication
• Readministering/desensitization
  • Consider risks and benefits of readministering therapy following severe hypersensitivity reaction (including anaphylaxis)
  • Patients may be rechallenged using slower infusion rates
  • In patients with severe hypersensitivity reactions, desensitization measures to this drug may be considered
• If readministered, ensure the patient tolerates the infusion
• If an infusion is tolerated, dose and rate may be increased to reach the approved recommended dosage
• Infusion-associated reactions
• Antihistamines, antipyretics, and corticosteroids can be given before administration to reduce the risk of IARs, however, IARs may still occur after receiving pretreatment
• Ensure appropriate medical support measures are readily available, including cardiopulmonary resuscitation equipment
• Discontinue infusion immediately if a severe reaction occurs; may attempt slower infusion rate if tolerated
• If mild or moderate IARs occur regardless of pretreatment, decreasing infusion rate or temporarily stopping infusion may ameliorate symptoms
• Assess benefits and risks of readministering therapy following severe IARs; may rechallenge using slower infusion rate; once infusion rate tolerated, may increase to reach recommended infusion rate
• Cardiorespiratory failure
  • 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 the infusion
• Perform more frequent monitoring of vitals
• Some patients may require prolonged observation times
• Embryofetal toxicity
  • Contraindicated during pregnancy
  • Based on findings from animal reproduction studies, Cipaglucosidase Alfa plus miglustat may cause embryofetal harm when administered to pregnant women
  • Verify pregnancy status in females of reproductive potential before initiating

Pregnancy and Lactation

• Contraindicated during pregnancy
• Based on findings from animal reproduction studies, it may cause embryofetal harm when administered to pregnant women
• Verify the pregnancy status in women of reproductive potential before initiating
• Contraception
  • Advise women of reproductive potential to use effective contraception during treatment and for at least 60 days after the last dose
• Infertility
  • Females: Based on preimplantation loss observed in female rats, it may impair human female fertility; it is unknown whether this preimplantation loss in female rats would be sustained if dosing were discontinued before mating
  • Males: Based on reversible increases in preimplantation loss in male rats, it may impair human male fertility
• Lactation
  • Data are not available regarding the presence of Cipaglucosidase Alfa, alone or in combination with miglustat, in human milk, effects on breastfed infants, or effects on milk production
  • Cipaglucosidase Alfa is present in animal milk; when a drug is present in animal milk, it will likely be present in human milk
  • Based on findings in animal studies, the use of Cipaglucosidase Alfa in combination with Miglustat may lead to serious adverse reactions in breastfed infants
  • Advise women that breastfeeding is not recommended during treatment