Xenpozyme
Xenpozyme (olipudase alfa or olipudase alfa-rpcp) is the only medication approved for use in adults and children with a rare, genetic condition called acid sphingomyelinase deficiency (ASMD). It's infused into the veins every 2 weeks by a healthcare provider. Because Xenpozyme (olipudase alfa) can put you at risk for serious allergic and infusion-related reactions, your provider will give you pretreatment medications before each infusion.
What is Xenpozyme (olipudase alfa)?
What is Xenpozyme (olipudase alfa) used for?
- Certain symptoms of acid sphingomyelinase deficiency (ASMD) in adults and children
How Xenpozyme (olipudase alfa) works
Xenpozyme (olipudase alfa) is an enzyme replacement therapy. If you have ASMD, your body can't make enough of an enzyme (protein) called acid sphingomyelinase (ASM). ASM is required to break down a specific type of fat in your cells called sphingomyelins. Without ASM, there's a build-up of sphingomyelins in your liver, spleen, lung, and brain. This can cause damage your organs.
Xenpozyme (olipudase alfa) serves as a replacement for ASM, which allows your body to break down the fat (sphingomyelins) in your cells.
Drug facts
Side effects of Xenpozyme (olipudase alfa)
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Common Side Effects
Adults
- Headache (54%)
- Cough (31%)
- Diarrhea (15%)
- Dizziness, tiredness, or other symptoms of low blood pressure (15%)
- Eye redness (15%)
Children
- Fever (100%)
- Cough (75%)
- Diarrhea (75%)
- Nasal congestion, runny nose, sneezing, itching (75%)
- Stomach pain (63%)
- Vomiting (50%)
- Headache (50%)
- Hives (50%)
- Nausea (38%)
- Rash (38%)
- Joint pain (38%)
- Itching (25%)
- Extreme tiredness (25%)
- Sore throat (25%)
Less Common Side Effects
Adults
- Skin redness
- Lack of energy
- Sore throat
- Trouble breathing
- Hives
- Small bumps on the skin
- Muscle pain
Children
- Dizziness, tiredness, or other symptoms of low blood pressure
- Severe allergic reaction
- Swelling at infusion site
- Fast heartbeat
- Throat swelling
Xenpozyme (olipudase alfa) serious side effects
Contact your healthcare provider immediately if you experience any of the following.
- Serious allergic reaction (anaphylaxis): difficulty breathing, hives, itching, fast heartbeat, tightness in the throat, nausea, stomach pain, dizziness, lightheadedness, swelling in the face or tongue
- Serious liver problems: swelling of your stomach, yellowing of your skin or the whites of your eyes, confusion, dark or brown urine
The following Xenpozyme (olipudase alfa) side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions or angioedema—skin rash, itching or hives, swelling of the face, eyes, lips, tongue, arms, or legs, trouble swallowing or breathing
- Infusion reactions—chest pain, shortness of breath or trouble breathing, feeling faint or lightheaded
- Liver injury—right upper belly pain, loss of appetite, nausea, light-colored stool, dark yellow or brown urine, yellowing skin or eyes, unusual weakness or fatigue
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
Pros and cons of Xenpozyme (olipudase alfa)
Pros
- Works well to improve lung function, make spleen and liver size smaller, and improve platelet count in adults and children
- Given once every 2 weeks
- Can be given at home if provider says it's safe to do so
Cons
- Brand name only, which can be expensive
- Only used for treating symptoms of ASMD and isn't a cure for ASMD
- The dose needs to be raised slowly to lower the risk for allergic reactions and side effects
Pharmacist tips for Xenpozyme (olipudase alfa)
- Make sure to mark your appointments on your calendar for when you need to see your provider and receive Xenpozyme (olipudase alfa) so you don't miss any doses. You'll typically receive an infusion every 2 weeks.
- If you accidentally miss a dose of Xenpozyme (olipudase alfa), talk with your provider right away about rescheduling your appointment. Depending on how many doses you missed, your provider might need to restart you at a lower dose.
- The entire Xenpozyme (olipudase alfa) infusion can take about 4 hours (including the time for premedication and post-infusion observation). You're recommended to wear comfortable clothes and have something with you, such as a book or a movie, to help pass the time.
- Xenpozyme (olipudase alfa) can cause serious allergic reactions. This can happen even if you've safely received this medication before. If you experience any allergic reaction, such as hives, tightness in your throat, or trouble breathing, let your nurse know right away. They'll need to stop Xenpozyme (olipudase alfa) and give you medical attention right away.
- You might experience infusion-related reactions up to 2 days after your Xenpozyme (olipudase alfa) infusion. Let your provider know right away if you experience headache, nausea, or rash, even after you're already home. You might need more medical attention.
- Xenpozyme (olipudase alfa) can cause problems during pregnancy, including harm to unborn babies. If you're a female who's able to become pregnant, you should use birth control during the Xenpozyme (olipudase alfa) treatment course and for at least 2 weeks after the treatment course is complete.
More tips for people administering Xenpozyme (olipudase alfa) at home:
- If your healthcare provider says it's safe for you to start receiving Xenpozyme (olipudase alfa) at home, you'll need to prepare your home environment. Read through this guide to prepare yourself for home infusions.
- You'll receive a large box of medical supplies that you'll need for Xenpozyme (olipudase alfa) infusions. After you receive the box, open it up and look through it to see that nothing is missing and that no medication has leaked or spilled. Don't shake box.
- Some items in your medical supplies box need to be stored in the refrigerator. Make sure you put them in the refrigerator right away after delivery.
- Xenpozyme (olipudase alfa) might be delivered straight to your home or to a specialty pharmacy nearby. Please check in with your provider so you know where to pick up your medication.
- If Xenpozyme (olipudase alfa) is getting delivered to your house, make sure you have space in your refrigerator to store it. It might be convenient to have a separate space, such as a mini-fridge, just to store Xenpozyme (olipudase alfa).
- On the day of your infusion, a trained nurse will come to your home to give you the Xenpozyme (olipudase alfa) infusion. The nurse will check your medical supplies and explain to you how the infusion process will go. They will give you pretreatment medication to lower your risk for serious allergic reactions and stay with you throughout the entire infusion.
- Make sure you have a sharps container or something made of heavy-duty plastic (e.g., empty milk carton, laundry detergent bottle) where the nurse can throw away needles. When your sharps container is almost full, close it tightly with the cap. For more information on how to throw away your sharps container safely and guidance for your specific state, visit the FDA's website.
Common questions about Xenpozyme (olipudase alfa)
ASMD is a rare, genetic condition. People with ASMD are missing a specific enzyme that helps break down a type of fat. As a result, they experience a build-up of fat and that leads to organ damage. Signs and symptoms of ASMD will depend on which organs are affected, but the most common are an enlarged stomach, cough, difficulty breathing, easily bruising or bleeding, stomach pain, and diarrhea.
ASMD is a genetic problem and Xenpozyme (olipudase alfa) only helps ease certain symptoms. Your provider will likely have you continue Xenpozyme (olipudase alfa) treatment as long as it's helping your condition and you're able to tolerate the side effects. Talk with your provider if you're not sure how long you need to receive Xenpozyme (olipudase alfa) treatment.
Let your provider know if you're thinking about receiving Xenpozyme (olipudase alfa) treatment at home. You'll receive the starting doses of Xenpozyme (olipudase alfa) at a clinic or infusion center. During this time, your provider will slowly raise your dose over the course of a few months. Once you've reached the maintenance dose, your provider will monitor you to see if you can tolerate this dose without any serious side effects. If so, your provider might approve of Xenpozyme (olipudase alfa) home infusions if they think it's safe for you. If approved, you'll need to prep your home for home infusions. A trained nurse will come to your home each time to give you Xenpozyme (olipudase alfa) and stay with you throughout the whole infusion.
Yes. Your provider will order blood tests to check you liver health a month before you start Xenpozyme (olipudase alfa). You'll also need to get blood tests to check your liver before each infusion, at least during the time when your provider is slowly raising your dose. This is because Xenpozyme (olipudase alfa) can cause liver damage and you might not experience any noticeable symptoms. If you're female, your provider will also give you a pregnancy test before you start Xenpozyme (olipudase alfa) to make sure you're not pregnant. This is because Xenpozyme (olipudase alfa) might cause serious harm to unborn babies if taken during pregnancy. If you're not sure whether your tests are scheduled, please check in with your provider.
Your provider might have prescribed you pre-medications (e.g., corticosteroid, antihistamine, or fever medication) to take before your infusion to help lower your risk for severe allergic and infusion reactions. Make sure to take your medications about 30 minutes before your Xenpozyme (olipudase alfa) dose.
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Risks and warnings for Xenpozyme (olipudase alfa)
Xenpozyme (olipudase alfa) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Severe allergic and infusion-related reactions
Xenpozyme (olipudase alfa) can cause serious, sometimes life-threatening, allergic reactions. These reactions can happen during any Xenpozyme (olipudase alfa) infusion, even if you've safely received infusions before. Because of this, your provider might give you pretreatment medications, such as diphenhydramine (Benadryl), acetaminophen (Tylenol), and corticosteroids, to lower your risk for these reactions. You'll also need to get your Xenpozyme (olipudase alfa) at a facility that has the equipment to give you medical care in case of an allergic reaction. If you experience a reaction, your provider might stop or pause the infusion, slow the infusion, or lower the dose of your medication. Get medical help right away if you experience symptoms of a serious allergic reaction, such as trouble breathing, tightness in your throat, hives and itching, and a fast heartbeat.
Xenpozyme (olipudase alfa) can also cause infusion-related reactions. Most people might experience these reactions during the first few months of treatment, when their provider is slowly raising their dose. These reactions typically happen within 2 days of the infusion. The pretreatment medications can lower your risk for these reactions. Let your provider know if you experience symptoms of infusion-related reactions, such as fever, headache, nausea, vomiting, and rash.
Liver problems
- Risk factors: History of liver problems
Xenpozyme (olipudase alfa) can cause liver damage. This usually occurs within 2 days of the infusion. Typically, you won't notice any symptoms. But your provider can tell whether or not you're having liver problems based on blood work. Because of this risk, your healthcare provider will order labs to check your liver health a month before you start Xenpozyme (olipudase alfa) treatment. You'll likely need to get labs before each infusion to make sure your liver is working properly. If you have liver damage, your provider might need to pause Xenpozyme (olipudase alfa) infusions to give your liver a chance to recover and to prevent further damage. Get medical attention right away if you notice swelling of your stomach, stomach pain, yellowing of your skin or the whites of your eyes, stool changes (black, tarry, or bloody stools), nausea, vomiting, confusion, or dark or brown urine.
Risk of harm in pregnancy
Xenpozyme (olipudase alfa) isn't recommended during pregnancy because of the potential harm it can cause to unborn babies (e.g., brain and other developmental problems). Your provider will ask you take a pregnancy test before you start Xenpozyme (olipudase alfa). Females who are able to get pregnant should use birth control during treatment and for at least 2 weeks after treatment with Xenpozyme (olipudase alfa). If you're pregnant or thinking of becoming pregnant, talk to your provider about the risks and benefits of using Xenpozyme (olipudase alfa) during pregnancy.
Xenpozyme (olipudase alfa) dosage
Typical dosage for Xenpozyme (olipudase alfa)
Your provider will determine your (or your child's) dose of Xenpozyme (olipudase alfa) based on body weight.
Xenpozyme (olipudase alfa) is given as an infusion into the veins (IV) every 2 weeks by a healthcare provider.
The typical starting dose is 0.03 mg/kg (for children up to 17 years) or 0.1 mg/kg (for adults). Your provider will raise the dose slowly over the course of a few months until you (or your child) reach the recommended maintenance dose of 3 mg/kg IV every 2 weeks.
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