Xenpozyme Side Effects

Generic name: olipudase alfa

Medically reviewed by Drugs.com. Last updated on Dec 18, 2024.

Note: This document provides detailed information about Xenpozyme Side Effects associated with olipudase alfa. Some dosage forms listed on this page may not apply specifically to the brand name Xenpozyme.

Applies to olipudase alfa: intravenous powder for solution.

Precautions

It is very important that your doctor check your or your child's progress at regular visits to make sure that this medicine is working properly. Blood tests may be needed to check for unwanted effects.

Using this medicine while you are pregnant can harm your unborn baby. If you are a woman who can get pregnant, your doctor may give you a pregnancy test before you start using this medicine to make sure you are not pregnant. Use an effective form of birth control during treatment with this medicine and for at least 14 days after the last dose. If you think you have become pregnant while using this medicine, tell your doctor right away.

This medicine may cause serious allergic reactions, including anaphylaxis and angioedema, which can be life-threatening and require immediate medical attention. Check with your doctor right away if you have chest tightness, cough, difficulty swallowing, dizziness, fast heartbeat, hives, itching, or skin rash, large, hive-like swelling on the face, eyelids, lips, tongue, throat, hands, legs, feet, or sex organs, puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue, trouble breathing, or unusual tiredness or weakness.

This medicine may cause infusion-related reactions, which can be life-threatening and require immediate medical attention. Tell your doctor right away if you start to have a fever, chills or shaking, dizziness, trouble breathing, itching or rash, lightheadedness or fainting after receiving this medicine.

Serious side effects of Xenpozyme

Along with its needed effects, olipudase alfa (the active ingredient contained in Xenpozyme) may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor or nurse immediately if any of the following side effects occur while taking olipudase alfa:

Other side effects of Xenpozyme

Some side effects of olipudase alfa may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects.

Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

For healthcare professionals

Applies to olipudase alfa: intravenous powder for injection.

General adverse events

The most frequently reported adverse reactions were headache, pyrexia, urticaria, nausea, vomiting, abdominal pain, myalgia, pruritus, and increased C-reactive protein. In 1 trial, treatment-related serious adverse reactions occurred in a higher percentage of pediatric patients than in adult patients.^[Ref]

Cardiovascular

• Common (1% to 10%): Palpitations, tachycardia, hypotension, hot flush, flushing, extrasystoles

In 1 trial, hypotension was reported in 15% of adult patients (n=13); hypotension and tachycardia were each reported in 13% of pediatric patients (n=8).

Extrasystoles occurred in an adult patient with history of cardiomyopathy.

Dermatologic

• Very common (10% or more): Urticaria (up to 21.7%), pruritus (10%)
• Common (1% to 10%): Angioedema, fixed eruption, rash, papular rash, macular rash, maculopapular rash, erythematous rash, pruritic rash, morbilliform rash, papule, macule, erythema

In 1 trial, erythema, urticaria, and papule were each reported in 8% of adult patients (n=13); urticaria, rash, and pruritus were reported in 50%, 38%, and 25% of pediatric patients (n=8), respectively. Rash included rash and erythema.

Gastrointestinal

• Very common (10% or more): Nausea (up to 20%), vomiting (up to 16.7%), abdominal pain (up to 15%)
• Common (1% to 10%): Diarrhea, upper abdominal pain, abdominal discomfort, gastrointestinal pain

In 1 trial, diarrhea was reported in 15% of adult patients (n=13); diarrhea, abdominal pain, vomiting, and nausea were reported in 75%, 63%, 50%, and 38% of pediatric patients (n=8), respectively. Abdominal pain included abdominal pain and upper abdominal pain.

Hepatic

• Common (1% to 10%): Hepatic pain, increased ALT, increased AST
• Frequency not reported: Elevated transaminase levels

Elevated transaminase levels (ranging from 3 to 14 times the upper limit of normal) were reported in 4 (13%) adult patients and 1 (13%) pediatric patient during the dose escalation phase in clinical trials.

Hypersensitivity

• Very common (10% or more): Hypersensitivity-related infusion-associated reactions (up to 26.7%)
• Common (1% to 10%): Anaphylactic reaction, hypersensitivity

Hypersensitivity-related infusion-associated reactions (including anaphylaxis) occurred in 26.7% patients (17.5% adult and 45% pediatric patients) in clinical trials. The most frequently reported hypersensitivity-related infusion-associated reaction symptoms were urticaria, pruritus, erythema, and rash.

Anaphylactic reactions were reported in 3 pediatric patients. An 18-month-old patient treated with this drug experienced an anaphylactic reaction during the sixth infusion in the dose escalation period. A 16-month-old patient with acid sphingomyelinase deficiency (ASMD) type A, treated with a version of this drug manufactured from a different process, experienced 2 anaphylactic reactions during the fifth and sixth infusions in the dose escalation period; the patient received an immune tolerance induction therapy prior to treatment. In the postmarketing setting, a 32-month-old patient experienced an anaphylactic reaction.

Hypersensitivity reactions (including anaphylaxis) occurred in a higher percentage of pediatric patients than in adult patients. Hypersensitivity reactions that were mild to moderate in severity occurred in 10 (33%) adult patients and 4 (50%) pediatric patients in clinical trials. Hypersensitivity reactions in adult patients included urticaria, pruritus, erythema, rash, erythematous rash, eczema, angioedema, and erythema nodosum. Hypersensitivity reactions in pediatric patients included urticaria, pruritus, rash, erythema, and localized edema. In 1 trial, anaphylactic reaction and hypersensitivity were each reported in 13% of pediatric patients (n=8).

Immunologic

• Very common (10% or more): Development of antidrug antibodies
• Frequency not reported: Immunoglobulin G (IgG) antidrug antibodies present, IgE antidrug antibodies present, neutralizing antibodies present

Overall, 16 of 40 (40%) adult patients and 13 of 20 (65%) pediatric patients treated with this drug developed treatment-emergent antidrug antibodies (ADA); the median time to seroconversion from first infusion was about 33 weeks in adult patients and 10 weeks in pediatric patients. Most ADA-positive patients (11 of 16 adult and 8 of 13 pediatric patients) had a low ADA response (up to 400) or reverted to ADA-negative. Of the ADA-positive patients, 4 of 16 adult and 5 of 13 pediatric patients had neutralizing antibodies that inhibited the activity of this drug; 6 patients developed neutralizing antibodies at a single time point and 3 patients had an intermittent response. A pediatric patient had a treatment boosted ADA response. Another pediatric patient had an anaphylactic reaction and developed IgE ADA and IgG ADA with a peak titer of 1600.

No effect of ADA was observed on pharmacokinetics and efficacy of this drug in adult and pediatric patients. There was a higher percentage of patients with treatment-emergent infusion-associated reactions (including hypersensitivity reactions) in patients who developed treatment-emergent ADA versus those who did not (75.9% versus 41.9%).

Local

• Common (1% to 10%): Catheter site pain, catheter site related reaction, catheter site pruritus, catheter site swelling
• Frequency not reported: Infusion-site swelling

In 1 trial, infusion-site swelling was reported in 13% of pediatric patients (n=8).

Musculoskeletal

• Very common (10% or more): Myalgia (up to 11.7%)
• Common (1% to 10%): Bone pain, arthralgia, back pain

In 1 trial, myalgia was reported in 8% of adult patients (n=13); arthralgia was reported in 38% of pediatric patients (n=8).

Nervous system

• Very common (10% or more): Headache (up to 31.7%)

In 1 trial, headache was reported in 54% of adult patients (n=13) and 50% of pediatric patients (n=8).

Ocular

• Common (1% to 10%): Ocular hyperemia, ocular discomfort, eye pruritus

In 1 trial, ocular hyperemia was reported in 15% of adult patients (n=13).

Other

• Very common (10% or more): Infusion-associated reactions (up to 65%), pyrexia (up to 25%), increased C-reactive protein (10%)
• Common (1% to 10%): Pain, chills, fatigue, asthenia, increased serum ferritin, abnormal C-reactive protein, increased body temperature
• Frequency not reported: Acute phase reaction

Infusion-associated reactions (IARs) were reported in 55% of adult and 65% of pediatric patients. IAR symptoms reported most frequently in adult patients were headache, nausea, urticaria, arthralgia, myalgia, pyrexia, pruritus, vomiting, and abdominal pain. IAR symptoms reported most frequently in pediatric patients were pyrexia, urticaria, vomiting, headache, nausea, and rash. IARs typically occurred between the time of infusion and 24 hours after infusion end.

In 1 trial, asthenia and abnormal C-reactive protein were each reported in 8% of adult patients (n=13); pyrexia, fatigue, and increased C-reactive protein were reported in 100%, 25%, and 13% of pediatric patients (n=8), respectively. Fatigue included fatigue and asthenia.

In 2 adult and 3 pediatric patients, IAR symptoms were associated with changes in laboratory parameters (e.g., C-reactive protein, ferritin value) indicative of acute phase reaction. Most of the acute phase reactions occurred at 48 hours post-infusion during the dose escalation period. Elevations of C-reactive protein, calcitonin, and interleukin 6, and reduction of serum iron were observed. The most common clinical symptoms associated with acute phase reactions were pyrexia, vomiting, and diarrhea. Acute phase reactions were managed similar to other IARs. In the postmarketing setting, 24 hours after receiving this drug at a higher than recommended initial dose, a 2-year-old male patient with ASMD experienced fever, respiratory distress, hypotension, and death.

Respiratory

• Common (1% to 10%): Pharyngeal edema, pharyngeal swelling, throat tightness, wheezing, larynx irritation, dyspnea, throat irritation

In 1 trial, cough, pharyngitis, dyspnea, and throat irritation were reported in 31%, 8%, 8%, and 8% of adult patients (n=13), respectively; cough, rhinitis, pharyngitis, and pharyngeal swelling were reported in 75%, 75%, 25%, and 13% of pediatric patients (n=8), respectively.

See also:

Frequently asked questions

• How effective is Xenpozyme?
• How does Xenpozyme work?

Further information

Xenpozyme side effects can vary depending on the individual. Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Note: Medication side effects may be underreported. If you are experiencing side effects that are not listed, submit a report to the FDA by following this guide.

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