Vimizim
Vimizim (elosulfase alfa) is an enzyme replacement therapy. It's FDA-approved for adults and children to treat a rare genetic problem called Morquio syndrome type IVA. Vimizim (elosulfase alfa) is infused through the veins once a week by a healthcare provider. Your provider will give you antihistamines before each dose to lower the risk of serious allergic reactions. Some common side effects of Vimizim (elosulfase alfa) include headache, nausea, and vomiting.
What is Vimizim (elosulfase alfa)?
What is Vimizim (elosulfase alfa) used for?
Mucopolysaccharidosis type IVA (MPS IVA or Morquio A syndrome) in adults and children ages 5 years and older
How Vimizim (elosulfase alfa) works
People with Morquio syndrome are missing an enzyme (protein) called GALNS, which is responsible for breaking down certain types of sugar called glycosaminoglycans (GAG). Without the enzyme, high levels of GAG can build up and cause problems in the heart, lungs, and bones.
Vimizim (elosulfase alfa) is an enzyme replacement therapy used to treat Morquio A syndrome by replacing missing GALNS in your body.
Drug Facts
What are the side effects of Vimizim (elosulfase alfa)?
Common Side Effects
- Fever (33%)
- Vomiting (31%)
- Headache (26%)
- Nausea (24%)
- Stomach pain (21%)
Other Side Effects
- Chills
- Tiredness
Serious Side Effects
- 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
Source: DailyMed
The following 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
- Severe back pain, numbness or weakness of the hands, arms, legs, or feet, loss of coordination, loss of bowel or bladder control
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 Vimizim (elosulfase alfa)
Cons
Each infusion takes at least 4 hours
Can experience serious allergic reactions up to 6 days after the dose
Pharmacist tips for Vimizim (elosulfase alfa)
Make sure to mark your appointments on your calendar for when you need to see your provider and receive Vimizim (elosulfase alfa) so you don't miss any doses.
The entire Vimizim (elosulfase alfa) infusion can take about 4 hours. You're recommended to get a good night's sleep, wear comfortable clothes, and have something with you (e.g., a book, tablet, or video game) to help pass the time. Also, make sure to plan ahead for this time by taking some time off of work or school or arrange for childcare.
You might experience an allergic reaction or infusion-related reaction during or after your Vimizim (elosulfase alfa) infusion. This can happen anywhere from 30 minutes to 6 days after you've started or received the infusion. You can still get allergic reactions even if you didn't get a reaction to a dose before. If you have any signs or symptoms of an allergic reaction, including trouble breathing or rash, get medical help right away so you can be treated as soon as possible.
Make sure to take any pre-medications (e.g., acetaminophen (Tylenol) and diphenhydramine (Benadryl)) before your infusion to prevent or ease any allergic reactions from Vimizim (elosulfase alfa). These are typically taken 30 minutes to 1 hour before your Vimizim (elosulfase alfa) dose.
It's not completely known if Vimizim (elosulfase alfa) is safe during pregnancy. Animal studies show babies born to mothers exposed to Vimizim (elosulfase alfa) at doses much higher than the typical dose used in humans were harmed. Additionally, Vimizim (elosulfase alfa) can get into breastmilk, which can reach breastfed babies and potentially cause side effects. If you're pregnant or breastfeeding and are receiving Vimizim (elosulfase alfa), it's recommended to enroll in the Morquio A Registry. The registry monitors you and your baby's health outcomes during treatment. Enrolled by contacting MARS@bmrn.com or by calling 1-800-983-4587.
Frequently asked questions about Vimizim (elosulfase alfa)
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What are the risks and warnings for Vimizim (elosulfase alfa)?
Vimizim (elosulfase 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
Vimizim (elosulfase alfa) can cause serious, sometimes life-threatening, allergic reactions. These reactions can happen during any Vimizim (elosulfase alfa) infusion, even if you've safely received infusions before. You might notice symptoms as early as 30 minutes after the start of the infusion, or as late as 6 days after the dose.
Because of this, your provider might give you pretreatment medications, such as diphenhydramine (Benadryl) or acetaminophen (Tylenol), to lower your risk for these reactions. You'll also need to get your Vimizim (elosulfase 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, cough, a fast heartbeat, and stomach-upset.
Sudden breathing problems
Risk factors: Fever | Breathing or lung problems
If you're sick with a fever or have a respiratory illness at the time of your appointment to receive Vimizim (elosulfase alfa), let your provider know. They might hold off on giving you your dose because you might be at higher risk of developing life-threatening complications from allergic reactions.
Let your provider know if you have sleep apnea or if you use oxygen or continuous positive airway pressure (CPAP) during sleep so they can make it available for you to use if you need it during or after your treatment. Let your provider know your full medical history before starting treatment with Vimizim (elosulfase alfa), since you might need to be monitored more carefully.
Spinal or cervical cord compression
Spinal or cervical cord compression (SCC) is symptom of worsening Morquio A syndrome. This is when your spinal or cervical cord is being compressed (or squeezed), which can lead to loss of feelings or nerve function in certain parts of your body. In clinical trials, both people who received Vimizim (elosulfase alfa) and placebo experienced this condition. Let your provider know if you experience symptoms of SCC, such as back pain, paralysis (loss of movement) in your arms or legs, and loss of bladder control. They can talk with you about appropriate next steps.
Vimizim (elosulfase alfa) dosage forms
Typical dosing for Vimizim (elosulfase alfa)
Your provider will determine the dose based on your body weight.
The typical dose is 2 mg/kg of body weight infused into the veins (intravenous or IV) over 3.5 to 4.5 hours once per week.
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References
Best studies we foundAmerican Lung Association. (2023). Six-minute walk test.
BioMarin Pharmaceutical Inc. (n.d.). Work with your healthcare team to set treatment goals before starting Vimizim® (elosulfase alfa).
BioMarin Pharmaceutical Inc. (2021). Vimizim- elosulfase alfa injection, solution, concentrate [package insert]. DailyMed.
Casale, J., et al. (2023). Biochemistry, glycosaminoglycans. StatPearls.
MedlinePlus. (2010). GALNS gene.
MedlinePlus (2019). Mucopolysaccharidosis type IV.
National Organization for Rare Disorders. (2019). Mucopolysaccharidosis IV.
Park, D. K., et al. (2022). Cervical spondylotic myelopathy (spinal cord compression). OrthoInfo.
Sanford, M., et al. (2014). Elosulfase alfa: First global approval. Drugs.
Schrover, R., et al. (2017). Minimal clinically important difference for the 6-min walk test: Literature review and application to Morquio A syndrome. Orphanet Journal of Rare Diseases.
U.S. Food and Drug Administration. (n.d.). Search orphan drug designations and approvals.
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