Elfabrio
Elfabrio (pegunigalsidase alfa or pegunigalsidase alfa-iwxj) is used to treat a rare genetic problem called Fabry disease. This medication is injected into the veins every 2 weeks by a healthcare provider. Side effects include headache and infusion-related reactions, such as nausea, chills, itchy or flushed skin, chest pain.
What is Elfabrio (pegunigalsidase alfa)?
What is Elfabrio (pegunigalsidase alfa) used for?
- Fabry disease in adults
How Elfabrio (pegunigalsidase alfa) works
Elfabrio (pegunigalsidase alfa) is an enzyme replacement therapy. It provides the body with an enzyme (protein) called alpha-galatosidase A.
In Fabry disease, your body isn't able to make the alpha-galatosidase A enzyme. This enzyme is important for breaking down fat (lipids) to help provide energy to your body. Without alpha-galatosidase A, fat builds up in the body and causes damage to many parts of the body, including the skin, kidneys, heart, and nervous system.
By replacing the missing alpha-galatosidase A enzyme, Elfabrio (pegunigalsidase alfa) helps lower fat buildup in the body for people with Fabry disease.
Drug facts
Side effects of Elfabrio (pegunigalsidase 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
- Infusion-related reactions (32%)
- Common cold symptoms (21%)
- Headache (21%)
- Diarrhea (19%)
- Fatigue (17%)
- Nausea (17%)
- Back pain (15%)
- Pain in arms or legs (15%)
- Stomach pain (12%)
Less Common Side Effects
- Allergic reaction
- Nerve pain
- Bloody urine
Elfabrio (pegunigalsidase alfa) serious side effects
Contact your healthcare provider immediately if you experience any of the following.
- Serious allergic reaction (e.g., anaphylaxis): difficulty breathing, tightness in the throat, hives, itching, fast heartbeat, nausea, dizziness, lightheadedness, swelling in the face or tongue
The following Elfabrio (pegunigalsidase alfa) side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
- Infusion reactions—chest pain, shortness of breath or trouble breathing, feeling faint or lightheaded
- Kidney injury (glomerulonephritis)—decrease in the amount of urine, red or dark brown urine, foamy or bubbly urine, swelling of the ankles, hands, or feet
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
- Diarrhea
- Fatigue
- Headache
- Nausea
- Runny or stuffy nose
- Sore throat
Pros and cons of Elfabrio (pegunigalsidase alfa)
Pros
- Might help improve kidney health in people with Fabry disease
- Injected every 2 weeks
Cons
- Not a cure for Fabry disease
- Hasn't been studied in children
Pharmacist tips for Elfabrio (pegunigalsidase alfa)
- Make sure to mark your appointments on your calendar for when you need to see your provider and get your Elfabrio (pegunigalsidase alfa) infusions. This helps you keep track of your appointments so you don't miss any doses. You'll typically receive an infusion every 2 weeks. If you miss an appointment, contact your provider's office as soon as possible to reschedule.
- Each Elfabrio (pegunigalsidase alfa) infusion will take at least 3 hours for the first few infusions. It might take longer if you experience a reaction to the infusion, since your provider will need to temporarily stop or slow down the infusion. You can bring a book, something to work on, or an electronic device to keep yourself occupied while you receive Elfabrio (pegunigalsidase alfa).
- You might experience infusion-related reactions when you're treated with Elfabrio (pegunigalsidase alfa). Let your provider or nurse know right away if you experience fever, chills, nausea, cough, dizziness, chest pain, or rash during the infusion or even when you're already home. You might need more medical attention.
- Elfabrio (pegunigalsidase 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 stop the Elfabrio (pegunigalsidase alfa) infusion and give you medical attention right away.
Common questions about Elfabrio (pegunigalsidase alfa)
Fabry disease can affect many parts of your body, causing symptoms like burning pain in the arms and legs, vision changes, fever, and kidney, heart, or stomach problems. To date, studies have measured the kidney health of people with Fabry disease taking Elfabrio (pegunigalsidase alfa). These studies suggest that Elfabrio (pegunigalsidase alfa) might be helpful in improving kidney health.
No. Elfabrio (pegunigalsidase alfa) is an IV infusion that's given only by a provider at their office or at an infusion center. This is because the provider has the proper resources, training, and technique to prepare and give the medication safely. While you can't give yourself the medication at home, it might be possible to have a trained provider come to your home for your Elfabrio (pegunigalsidase alfa) infusions. Discuss with your provider if home infusions of this medication is an option for you.
Typically, your Elfabrio (pegunigalsidase alfa) infusion will take at least 3 hours for the first four to six infusions. If you don't have any bothersome reactions to the medication during the first several infusions, your provider might adjust how fast you get your following infusions so that they take less time. Keep in mind that your Elfabrio (pegunigalsidase alfa) infusion will need to take 1.5 hours at the minimum. If you end up getting infusion-related reactions during your infusion, your provider might slow or temporarily stop the Elfabrio (pegunigalsidase alfa) infusion.
Your provider might give you certain medications to take before your Elfabrio (pegunigalsidase alfa) infusion. These medications help lower your risk for or the severity of infusion reactions from Elfabrio (pegunigalsidase alfa). The medications your provider might give you before your infusion typically include corticosteroids, antihistamines, or medications to relieve fever or pain, such as acetaminophen (Tylenol). Your provider might prescribe these medications for you to take at home before your Elfabrio (pegunigalsidase alfa) infusion, or you might receive them at your appointments. If you're prescribed these medications to take at home, make sure to take them about 30 minutes before your Elfabrio (pegunigalsidase alfa) dose. That's generally the amount of time it takes for most of these medications to start working.
Elfabrio (pegunigalsidase alfa) hasn't been studied in pregnant women, so it's not known how safe the medication is for you or your unborn baby. In animal pregnancy studies, babies born to mothers exposed to Elfabrio (pegunigalsidase alfa) at almost 4-times the typical dose used in humans weren't harmed. If you're pregnant or thinking of becoming pregnant, talk to your provider about the risks and benefits of continuing Elfabrio (pegunigalsidase alfa) treatment. It's encouraged to report a pregnancy that happens during treatment to the manufacturer (Chiesi USA) by calling 1-888-661-9260. The manufacturer has a pregnancy safety study that keeps track of health outcomes of people and babies exposed to Elfabrio (pegunigalsidase alfa) during pregnancy.
Elfabrio (pegunigalsidase alfa) hasn't been studied in women who are breastfeeding. For this reason, it's not known whether the medication is found in breast milk, how it could affect your milk supply, and the effects it could have on your breastfed baby. If you're breastfeeding, talk to your provider about the risks and benefits of continuing Elfabrio (pegunigalsidase alfa) treatment.
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Risks and warnings for Elfabrio (pegunigalsidase alfa)
Elfabrio (pegunigalsidase 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.
Allergic reactions
- Risk factors: People with antibodies against Elfabrio (pegunigalsidase alfa) in their blood
Elfabrio (pegunigalsidase alfa) can cause severe and sometimes life-threatening allergic reactions, including anaphylaxis. Get medical attention right away if you experience symptoms of an allergic reaction, such as hives, facial swelling, trouble breathing, tightness in your throat, and a fast heartbeat.
Infusion-related reactions
- Risk factors: People with antibodies against Elfabrio (pegunigalsidase alfa) in their blood
Elfabrio (pegunigalsidase alfa) can cause infusion-related reactions. These reactions include nausea, vomiting, chills, itching, flushing, chest pain, dizziness, headache, weakness, shortness of breath, and muscle ache, among others. They can happen during any Elfabrio (pegunigalsidase alfa) infusion or up to a day after you've gone home. To lower the severity of these reactions and your overall risk, your provider might give you pretreatment medications, such as diphenhydramine (Benadryl), acetaminophen (Tylenol), and corticosteroids. If you experience a reaction while you're getting an infusion, your provider might stop, pause, or slow the infusion. Get medical help as soon as possible if you experience symptoms of a serious reaction, such as trouble breathing, tightness in your throat, hives, and itching.
Elfabrio (pegunigalsidase alfa) dosage
Typical dosage for Elfabrio (pegunigalsidase alfa)
Your healthcare provider will calculate the right dose for you based on your body weight. The typical dose is 1 mg/kg of body weight injected into the veins (intravenous or IV) every 2 weeks.
What are alternatives to Elfabrio (pegunigalsidase alfa)?
Elfabrio (pegunigalsidase alfa) images
