Olipudase Alfa

Olipudase Alfa is a prescription medication used for the treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD).

• Olipudase Alfa is available under the following different brand names: Xenpozyme, olipudase Alfa-rpcp

Common side effects of Olipudase Alfa include:

• headache
• cough
• diarrhea
• low blood pressure (hypotension)
• eye redness
• fever
• runny nose
• abdominal pain
• nausea
• vomiting
• hives
• rash
• joint pain
• itching
• fatigue
• sore throat

Serious side effects of Olipudase Alfa include:

• hives
• itching or skin rash
• blurred vision
• body aches or pain
• chest tightness
• confusion
• dizziness
• faintness
• light-headedness
• dryness or soreness of the throat
• fast, pounding, or irregular heartbeat or pulse

Rare side effects of Olipudase 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, very 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 and pediatric dosage

Injection, lyophilized powder for reconstitution

• 20 mg/single-dose vial

Acid sphingomyelinase deficiency

Adult and pediatric dosage

• Weight-based dosing
  • Dose escalation and maintenance phases based on body weight.
  • BMI 30 and below: Dose based on actual body weight (kg).
  • BMI above 30: Dose based on adjusted body weight.
  • Calculate an adjusted body.
  • Adjusted body weight (kg) = (actual height in m)2 ×30
• Dose escalation
  • Gradual dose escalation is used to reduce hypersensitivity and infusion-associated reactions or elevated transaminase levels.
  • Dose 1 (day 1/week 0): 0.1 mg/kg IV
  • Dose 2 (week 2): 0.3 mg/kg IV
  • Dose 3 (week 4): 0.3 mg/kg IV
  • Dose 4 (week 6): 0.6 mg/kg IV
  • Dose 5 (week 8): 0.6 mg/kg IV
  • Dose 6 (week 10): 1 mg/kg IV
  • Dose 7 (week 12): 2 mg/kg IV
  • Dose 8 (week 14): 3 mg/kg IV (recommended maintenance dose)
  • Note: The dose escalation phase includes the first 3 mg/kg dose.
• Maintenance phase
  • 3 mg/kg IV every 2 weeks.

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, healthcare provider, or pharmacist first.

• Olipudase Alfa has no noted severe interactions with any other drugs.
• Olipudase Alfa has no noted serious interactions with any other drugs.
• Olipudase Alfa has no noted moderate interactions with any other drugs.
• Olipudase 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

• None

Effects of drug abuse

• None

Short-Term Effects

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

Long-Term Effects

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

Cautions

• May cause fetal harm
• Hypersensitivity reactions including anaphylaxis
• Consider pretreating with antihistamines, antipyretics, and/or corticosteroids before infusions
• Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available during the drug’s administration
• If a severe hypersensitivity reaction (eg, anaphylaxis) occurs, discontinue immediately and initiate appropriate medical treatment.
• Following severe hypersensitivity reactions (including anaphylaxis), consider the risks and benefits of readministering the drug
• If a patient has been rechallenged using slower infusion rates at a dosage lower than the recommended dosage, a tailored desensitization procedure to therapy may be considered
• If the decision is made to readminister, ensure the patient tolerates infusion; if tolerated, dosage (dose and/or rate) may be increased to reach the recommended dosage
• If a mild or moderate reaction occurs, the infusion rate may be slowed or temporarily withheld, and/or the dose reduced
• Infusion-associated reactions
  • Antihistamines, antipyretics, and/or corticosteroids may be given before administration to reduce the risk for infusion-associated reactions (IARs); however, IARs may still occur in patients after receiving pretreatment
  • If severe IARs occur, discontinue immediately and initiate appropriate medical treatment
  • Consider the risks and benefits of readministering the drug following severe IARs
  • If a mild or moderate IAR occurs, slow down or temporarily withhold the infusion rate and/or reduce the dose
• Elevated transaminases levels
  • May be associated with elevated transaminases (ALT, AST, or both) within 24-48 hours after infusion
  • Assess ALT and AST within 1 month before initiating, within 72 hours before any infusion during dose escalation, which includes the first 3 mg/kg dose, or before the next scheduled infusion upon resuming the treatment following a missed dose
  • If the baseline or pre-infusion transaminase level (during the dose escalation phase) is above 2×ULN, repeat transaminase levels within 72 hours after infusion
  • If pre-infusion transaminase levels are elevated above baseline and 2× ULN and more before the next scheduled administration, either reduce the dose (repeat prior lower dose or reduce the dose) or temporarily withhold until transaminase levels return to baseline
  • Continued transaminase testing is recommended as part of routine clinical management upon reaching the recommended maintenance dose

Pregnancy and Lactation

• Based on findings from animal reproduction studies, olipudase Alfa may cause embryofetal harm when administered to pregnant women
• Dosage initiation or escalation, at any time during pregnancy, is not recommended as it may lead to elevated sphingomyelin metabolite levels that may increase the risk for fetal malformations
• However, the decision to continue or discontinue maintenance dosing in pregnancy should consider the woman’s need for the drug and potential drug-related risks to the fetus and potential adverse outcomes from untreated maternal acid sphingomyelinase deficiency (ASMD).
• Contraception
  • Verify pregnancy status in women of reproductive potential before initiating olipudase Alfa
  • Advise women of reproductive potential to use effective contraception during treatment and for 14 days after the last dose if Olipudase Alfa is discontinued
• Lactation
  • Data are not available on the presence of olipudase Alfa in human milk, its effects on breastfed infants, or milk production
  • The drug is present in animal milk; when a drug is present in animal milk, the drug will likely be present in human milk