Olipudase alfa

What is Olipudase alfa?

• Olipudase alfa (Xenpozyme) is a hydrolytic lysosomal sphingomyelin-specific enzyme consisting of 570 amino acids produced in a Chinese hamster ovary cell line by recombinant DNA technology.

What are the uses of this medicine?

• Olipudase alfa (Xenpozyme) is used for treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) (Niemann-Pick disease type A) in adult and pediatric patients.

How does this medicine work?

• Acid sphingomyelinase deficiency (ASMD) is a lysosomal storage disease that results from reduced activity of the enzyme acid sphingomyelinase (ASM), caused by pathogenic variants in the sphingomyelin phosphodiesterase 1 gene.
• ASM degrades sphingomyelin to ceramide and phosphocholine.
• The deficiency of ASM causes an intra-lysosomal accumulation of sphingomyelin (as well as cholesterol and other cell membrane lipids) in various tissues.
• Xenpozyme provides an exogenous source of ASM.
• Xenpozyme is not expected to cross the blood-brain barrier or modulate the CNS manifestations of ASMD.

Who Should Not Use this medicine ?

• This medicine have no usage limitations.

What drug interactions can this medicine cause?

Tell your doctor and pharmacist what other prescription and nonprescription medications, vitamins, nutritional supplements, and herbal products you are taking or plan to take.

Is this medicine FDA approved?

• Olipudase alfa was approved for medical use in the United States in August 2022.
• The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.

How should this medicine be used?

• Prior to initiating treatment, verify pregnancy status in females of reproductive potential and obtain baseline transaminase levels.
• Consider pretreating with antihistamines, antipyretics, and/or corticosteroids.

Recommended dosage: Recommended Dosage in Adult Patients:

• Recommended starting dose is 0.1 mg/kg administered as an intravenous infusion.

Maintenance Phase:

• The recommended maintenance dosage of Xenpozyme in adults is 3 mg/kg via intravenous infusion every 2 weeks.

Recommended Dosage in Pediatric Patients:

• The recommended starting dose of Xenpozyme in pediatric patients is 0.03 mg/kg.

Maintenance Phase:

• The recommended maintenance dosage of Xenpozyme in pediatric patients is 3 mg/kg via intravenous infusion every 2 weeks.

Administration:

• Xenpozyme (olipudase alfa-rpcp) for injection is supplied as a sterile, preservative-free, white to off-white lyophilized powder for reconstitution and dilution to be administered via intravenous infusion.

What are the dosage forms and brand names of this medicine?

This medicine is available in fallowing doasage form:

• As injection: 4 mg or 20 mg of olipudase alfa-rpcp as a lyophilized powder in a single-dose vial for reconstitution.

This medicine is available in fallowing brand namesː

• Xenpozyme

What side effects can this medication cause?

The most common side effects of this medicine may include: In adult patients:

• headache, cough, diarrhea, hypotension, and ocular hyperemia.

In pediatric patients:

• pyrexia, cough, diarrhea, rhinitis, abdominal pain, vomiting, headache, urticaria, nausea, rash, arthralgia, pruritus, fatigue, and pharyngitis.

Less common, but serious side effects may include:

• Hypersensitivity Reactions Including Anaphylaxis
• Infusion-Associated Reactions
• Elevated Transaminase Level

What special precautions should I follow?

• Advise the patient and caregiver that reactions related to the infusion may occur during and after Xenpozyme treatment, including anaphylactic reactions, other serious or severe hypersensitivity reactions, and IARs. Inform the patient and caregiver of the signs and symptoms of hypersensitivity reactions and IARs and to seek medical care should signs and symptoms occur.
• Xenpozyme may cause embryo-fetal harm. Advise the pregnant female and females of reproductive potential of the potential risk to the fetus. Advise a female patient and caregiver to inform their healthcare provider of a known or suspected pregnancy.
• Advise a female of reproductive potential to use effective contraception during treatment and for 14 days after the last dose if Xenpozyme is discontinued.
• Antihistamines, antipyretics, and/or corticosteroids may be given prior to Xenpozyme administration to reduce the risk of infusion-associated reactions (IARs). However, IARs may still occur in patients after receiving pretreatment. If severe IARs occur, discontinue Xenpozyme and initiate appropriate medical treatment.
• Xenpozyme may be associated with elevated transaminases (ALT, AST, or both) within 24 to 48 hours after infusion. Assess ALT and AST within one month prior to initiation of Xenpozyme, within 72 hours prior to any infusion during dose escalation, or prior to the next scheduled Xenpozyme infusion upon resuming treatment following a missed dose.
• There are no data on the presence of olipudase alfa-rpcp in human milk, the effects on the breastfed infant, or the effects on milk production. Olipudase alfa-rpcp is present in animal milk.

What to do in case of emergency/overdose?

• In case of overdose, call the poison control helpline of your country. In the United States, call 1-800-222-1222.

• Overdose related information is also available online at poisonhelp.org/help.
• In the event that the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services. In the United States, call 911.

Can this medicine be used in pregnancy?

• Based on findings from animal reproduction studies, Xenpozyme may cause embryo-fetal harm when administered to a pregnant female.
• Xenpozyme 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 of fetal malformations.
• Xenpozyme may cause embryo-fetal harm when administered during the first trimester of pregnancy.
• Verify the pregnancy status in females of reproductive potential prior to initiating Xenpozyme.
• Advise females of reproductive potential to use effective contraception during treatment and for 14 days after the last dose if Xenpozyme is discontinued.

Can this medicine be used in children?

• The safety and effectiveness of Xenpozyme for the treatment of non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) have been established in pediatric patients down to birth.

What are the active and inactive ingredients in this medicine?

Active ingredient:

• olipudase alfa-rpcp

Inactive ingredients:

• sodium phosphate, dibasic, heptahydrate
• sodium phosphate, monobasic, monohydrate
• sucrose
• methionine

Who manufactures and distributes this medicine?

Manufactured by:

• Genzyme Corporation
• 450 Water Street
• Cambridge, MA

What should I know about storage and disposal of this medication?

• Store refrigerated at 2°C to 8°C (36°F to 46°F).
• Do not freeze.
• For storage of reconstituted and diluted solution.

• Dailymed label info on Olipudase alfa
• FDA Olipudase alfa

Olipudase alfa Resources
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