Work with your healthcare team to set treatment goals before starting VIMIZIM® (elosulfase alfa)

It is important to establish an overall management plan with your doctor

Morquio A is heterogeneous, meaning it affects each person differently. An overall management plan can help you and your doctor develop realistic treatment expectations.

While some individuals will experience an improvement in endurance, preventing further decline is also a measure of success. Before you start treatment, your doctor should work with you to determine an individual plan. Your doctor may also conduct assessments before you start treatment.

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It is important to establish an overall management plan with your doctor

Morquio A is heterogeneous, meaning it affects each person differently. An overall management plan can help you and your doctor develop realistic treatment expectations.

While some individuals will experience an improvement in endurance, preventing further decline is also a measure of success. Before you start treatment, your doctor should work with you to determine an individual plan. Your doctor may also conduct assessments before you start treatment.

 

For many patients, the following assessments are used:

  • Endurance

  • 6-minute walk test (6MWT)

    measures how far you can walk in 6 minutes

  • The timed 25-foot walk test (T25FWT)

    is used for people who have difficulty walking


  • Respiratory

  • Respiratory forced vital capacity (FVC)

    measures the volume of air that can forcibly be exhaled after taking the deepest breath possible

  • Forced expiratory volume (FEV1)

    measures the maximum volume of air that can be forcibly exhaled in 1 second

  • Maximum voluntary ventilation (MVV)

    measures the maximum amount of air that can be inhaled and exhaled in 1 minute

For many patients, the following assessments are used:

  • Endurance

  • 6-minute walk test (6MWT)

    measures how far you can walk in 6 minutes

  • The timed 25-foot walk test (T25FWT)

    is used for people who have difficulty walking


  • Respiratory

  • Respiratory forced vital capacity (FVC)

    measures the volume of air that can forcibly be exhaled after taking the deepest breath possible

  • Forced expiratory volume (FEV1)

    measures the maximum volume of air that can be forcibly exhaled in 1 second

  • Maximum voluntary ventilation (MVV)

    measures the maximum amount of air that can be inhaled and exhaled in 1 minute

  • Endurance

    The 6-minute walk test (6MWT)
    measures how far you can walk in 6 minutes 

    The timed 25-foot walk test (T25FWT)
     
    is used for people who have difficulty walking


  • Respiratory

    Respiratory Forced vital capacity (FVC)
    measures the volume of air that can forcibly be exhaled after taking the deepest breath possible

    Forced expiratory volume (FEV1)
    measures the maximum volume of air that can be forcibly exhaled in 1 second

    Maximum voluntary ventilation (MVV)
    measures how much air can be inhaled and exhaled in 1 minute

Partner with your healthcare team to discuss disease assessments and the development of an individualized management plan that is right for you.

Partner with your healthcare team to discuss disease assessments and an individualized management plan that is right for you.

INDICATION
VIMIZIM® (elosulfase alfa) is indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).

IMPORTANT SAFETY INFORMATION

Life-threatening allergic reactions, known as anaphylaxis, can occur during VIMIZIM® (elosulfase alfa) infusions. Typical signs of anaphylaxis include cough, rash, throat tightness, hives, flushing, changes in skin color, low blood pressure, shortness of breath, chest pain, and gastrointestinal symptoms such as nausea, abdominal pain, retching, and vomiting. Contact your doctor or get medical help right away if these symptoms occur during or after VIMIZIM infusions. If you have a respiratory illness, you may be at risk for a sudden worsening of your condition, and you may require additional monitoring.

VIMIZIM is a prescription medicine. Before treatment with VIMIZIM, it is important to discuss your medical history with your doctor. Tell your doctor if you are sick or taking any medication and if you are allergic to any medicines. Also tell your doctor if you are pregnant, planning to become pregnant, or are a nursing mother. Your doctor will decide if VIMIZIM is right for you. If you have questions or would like more information about VIMIZIM, contact your doctor.

Anaphylaxis can occur during any VIMIZIM infusion and up to three hours after any infusion, and hypersensitivity reactions have been observed as early as 30 minutes from the start of infusion but as late as six days after infusion.

Serious and severe reactions can happen with VIMIZIM treatment, including life-threatening allergic reactions (anaphylaxis), hives, swelling, cough, shortness of breath, and flushing. You should receive medication such as antihistamines before VIMIZIM infusions to reduce the risk of reactions. If a reaction occurs, the infusion should be slowed or stopped and you may be given additional medication. If a severe reaction occurs, the infusion should be stopped immediately and you will receive appropriate medical treatment.

If you have acute febrile or respiratory illness at the time of VIMIZIM infusion you may be at higher risk of life-threatening complications from hypersensitivity reactions. If you use supplemental oxygen or continuous positive airway pressure (CPAP) you should have it available during your infusion in the event of a sudden reaction, or extreme drowsiness/sleep from antihistamines.

Spinal cord damage may occur due to the natural MPS IVA disease process. Signs of spinal cord injury include back pain, numbness and paralysis, and loss of bladder and bowel control. Contact your doctor immediately if you develop any of these symptoms.

The most common side effects reported during VIMIZIM infusions included fever, vomiting, headache, nausea, abdominal pain, chills, and fatigue. These are not all of the possible side effects with VIMIZIM. Talk to your doctor if you have any symptoms that bother you or that do not go away.

Call your doctor for medical advice about side effects. You may report side effects to BioMarin Pharmaceutical Inc. at 1-866-906-6100, or FDA at 1-800-FDA-1088 or go to www.fda.gov/medwatch.

For more information, call BioMarin RareConnections at 1-866-906-6100.

Please see full Prescribing Information, including important warning.