VIMIZIM® (elosulfase alfa) works at a cellular level to help with deficient enzyme activity
VIMIZIM® (elosulfase alfa) works at a cellular level to help with deficient enzyme activity
How VIMIZIM® (elosulfase alfa) works
VIMIZIM® (elosulfase alfa) works at the cellular level in your body
People born with Morquio A are missing, or don’t have enough of, an enzyme known as N–acetylgalactosamine–6 sulfatase, or GALNS, which breaks down and recycles glycosaminoglycans, or GAGs, a type of cellular waste. VIMIZIM® (elosulfase alfa) can help replace the deficient GALNS to help restore some cell function.

How VIMIZIM® (elosulfase alfa) works
VIMIZIM® (elosulfase alfa) works at the cellular level in your body
People with Morquio A do not make enough of a specific enzyme, GALNS, which breaks down and recycles GAGs, a type of cellular waste. VIMIZIM® (elosulfase alfa) can help replace the deficient GALNS to help restore some cell function.

In people with Morquio A, GAGs build up in cells throughout the tissues and organs of the body, potentially causing serious problems.

A weekly infusion of VIMIZIM helps replace the deficient GALNS enzyme your body needs to help reduce the buildup of certain GAGs.

Taking VIMIZIM every week can help people with Morquio A reduce the buildup of certain GAGs.
VIMIZIM improved endurance with long-lasting benefits
VIMIZIM improved endurance at 6 months
aA baseline is a measurement of a person’s performance before they begin treatment in a clinical trial. To participate in the clinical trial for VIMIZIM, people had to be able to walk more than 30 meters but less than 325 meters in 6 minutes.
bA placebo is a harmless substance that does not contain any medicine.
Patients in the VIMIZIM once-weekly treatment arm saw a 23.9% mean improvement in 6MWT over baseline.
In a 24-week clinical trial, people who received VIMIZIM infusions every week improved their endurance as measured by the 6-minute walk test (6MWT). The number of people included in the study was intentionally chosen to see the effect of VIMIZIM on the 6MWT and the 3-minute stair climbing test. Other tests or measures were assessed to see if they might be valid for future study.
- This graph shows that by the end of a 24-week trial, people who took VIMIZIM once a week walked an average of 23.9% farther than at baseline
- People who took VIMIZIM every other week did not show much improvement, and their results were similar to the people who took a placebo
Maintaining weekly infusions is essential to making the most out of your treatment
Talk to your doctor about your individual VIMIZIM treatment goals
Knowing what to expect from your treatment with VIMIZIM can help you know what success looks like. For some people, this might mean that the impact of Morquio A becomes stabilized. Work with your doctor to develop treatment goals that are right for you.
Side effects
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.
Serious and severe reactions were associated with VIMIZIM, including hypersensitivity reactions as well as life-threatening allergic reactions (anaphylaxis).
- In clinical trials, 18.7% of patients treated with VIMIZIM experienced hypersensitivity reactions and 7.7% experienced anaphylaxis
Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.
VIMIZIM can provide long-lasting benefits
A 120-week extension study showed that the benefits of VIMIZIM can be maintained over the long term and are consistent with the results of the primary study.
In the 2-year extension study (MOR-005), all patients transitioned over time to receive VIMIZIM as a once-weekly treatment. The results of the treated patient group could not be compared to placebo since the study did not include a placebo group.
Improved endurance
Results from the long-term extension study showed a 32.9-meter mean increase in the 6-minute walk test (6MWT) compared to baseline.
Helped lungs work better
Breathing function as measured by pulmonary function tests improved for patients in the long-term extension study compared to baseline.
- There was a 9.2% mean increase in FVC compared with baseline
- There was an 8.8% mean increase in FEV1 compared with baseline
FEV1, maximum volume of air that can be forcibly blown out in 1 second; FVC, volume of air that can forcibly be exhaled from the lungs after taking the deepest breath possible.
Side effects
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.
Serious and severe reactions were associated with VIMIZIM, including hypersensitivity reactions as well as life-threatening allergic reactions (anaphylaxis).
- In clinical trials, 18.7% of patients treated with VIMIZIM experienced hypersensitivity reactions and 7.7% experienced anaphylaxis
Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.
VIMIZIM improved endurance with long-lasting benefits
VIMIZIM improved endurance at 6 months
In a 24-week clinical trial, people who received VIMIZIM infusions every week improved their endurance as measured by the 6-minute walk test (6MWT). The number of people included in the study was intentionally chosen to see the effect of VIMIZIM on the 6MWT and the 3-minute stair climbing test. Other tests or measures were assessed to see if they might be valid for future study.
aA baseline is a measurement of a person’s performance before they begin treatment in a clinical trial. To participate in the clinical trial for VIMIZIM, people had to be able to walk more than 30 meters but less than 325 meters in 6 minutes.
bA placebo is a harmless substance that does not contain any medicine.
Patients in the VIMIZIM once-weekly treatment arm saw a 23.9% mean improvement in 6MWT over baseline
- This graph shows that by the end of a 24-week trial, people who took VIMIZIM once a week walked an average of 23.9% farther than at baseline
- People who took VIMIZIM every other week did not show much improvement, and their results were similar to the people who took a placebo
Maintaining weekly infusions is essential to making the most out of your treatment
Talk to your doctor about your individual VIMIZIM treatment goals
Knowing what to expect from your treatment with VIMIZIM can help you know what success looks like. For some people, this might mean that the impact of Morquio A becomes stabilized. Work with your doctor to develop treatment goals that are right for you.
Side effects
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.
Serious and severe reactions were associated with VIMIZIM, including hypersensitivity reactions as well as life-threatening allergic reactions (anaphylaxis).
- In clinical trials, 18.7% of patients treated with VIMIZIM experienced hypersensitivity reactions and 7.7% experienced anaphylaxis
Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.
VIMIZIM can provide long-lasting benefits
A 120-week extension study showed that the benefits of VIMIZIM can be maintained over the long term and are consistent with the results of the primary study.
In the 2-year extension study (MOR-005), all patients transitioned over time to receive VIMIZIM as a once-weekly treatment. The results of the treated patient group could not be compared to placebo since the study did not include a placebo group.
Improved endurance
Results from the long-term extension study showed a 32.9-meter mean increase in the 6-minute walk test (6MWT) compared to baseline.
Helped lungs work better
Breathing function as measured by pulmonary function tests improved for patients in the long-term extension study compared to baseline.
- There was a 9.2% mean increase in FVC compared with baseline
- There was an 8.8% mean increase in FEV1 compared with baseline
FEV1, maximum volume of air that can be forcibly blown out in 1 second; FVC, volume of air that can forcibly be exhaled from the lungs after taking the deepest breath possible.
Side effects
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.
Serious and severe reactions were associated with VIMIZIM, including hypersensitivity reactions as well as life-threatening allergic reactions (anaphylaxis).
- In clinical trials, 18.7% of patients treated with VIMIZIM experienced hypersensitivity reactions and 7.7% experienced anaphylaxis
Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.
VIMIZIM® (elosulfase alfa) improved endurance at 6 months
In a 24-week clinical trial, people who received VIMIZIM infusions every week improved their endurance as measured by the 6-minute walk test (6MWT). The number of people included in the study was intentionally chosen to see the effect of VIMIZIM on the 6-minute walk test and the 3-minute stair climbing test. Other tests or measures were assessed to see if they might be valid for future study.
aA baseline is a measurement of a person’s performance before they begin treatment in a clinical trial. To participate in the clinical trial for VIMIZIM, people had to be able to walk more than 30 meters but less than 325 meters in 6 minutes
bA placebo is a harmless substance that does not contain any medicine.
- This graph shows that by the end of a 24-week trial, people who took VIMIZIM once a week walked an average of 23.9% farther than at baseline
- People who took VIMIZIM every other week did not show much improvement, and their results were similar to the people who took a placebo
Serious and severe reactions were associated with VIMIZIM, including hypersensitivity reactions as well as life-threatening allergic reactions (anaphylaxis). In clinical trials, 18.7% of patients treated with VIMIZIM experienced hypersensitivity reactions and 7.7% experienced anaphylaxis.
Maintaining weekly infusions is essential to making the most out of your treatment
VIMIZIM® (elosulfase alfa) also showed positive results across many efficacy measures
Although statistical significance was only observed in the 6MWT results, favorable improvement was seen in the majority of exploratory efficacy endpoints.
ADDITIONAL ENDPOINTS: FEV1 favors VIMIZIM (59:58) / 1.82 (-5.52, 9.17), MVV favors VIMIZIM (59:58) / 10.31 (-1.79, 22.42), Growth rate favors VIMIZIM (40:44) / 0.39 (-0.08, 0.87), Z-score height favors VIMIZIM (40:44) / 0.14 (-0.03, 0.31), MPS HAQ caregiver favors VIMIZIM (58:57) / 0.85 (-1.14, 2.85), MPS HAQ mobility favors VIMIZIM (59:57) / 0.26 (-0.28, 0.80), MPS HAQ self-care favors Placebo (59:57) / -0.07 (-0.47, 0.33).
FEV1=forced expiratory volume in 1 second; MVV=maximum voluntary ventilation; Z-score height=how far a measurement deviates from the average; MPS HAQ caregiver=caregiver-completed MPS Health Assessment Questionnaire; MPS HAQ mobility=patient-completed MPS Health Assessment Questionnaire on mobility; MPS HAQ self-care=patient-completed MPS Health Assessment Questionnaire on self-care.
Talk to your doctor about your individual VIMIZIM treatment goals
Knowing what to expect from your treatment with VIMIZIM can help you know what “success” looks like. For some people, this might mean that the impact of Morquio A becomes stabilized. Work with your doctor to come up with treatment goals that are right for you.
See the side effects of VIMIZIM
VIMIZIM® (elosulfase alfa) can provide long-lasting benefits
A 120-week extension study showed that the benefits of VIMIZIM® (elosulfase alfa) can be maintained over the long term and are consistent with the results of the primary study.
In the 2-year extension study (MOR-005), all patients transitioned over time to receive VIMIZIM as a once-weekly treatment. The results of the treated patient group could not be compared to placebo since the study did not include a placebo group.

Improved endurance
Results from the long-term extension study showed a 32.9-meter mean increase in the 6-minute walk test (6MWT) compared to baseline.

Helped lungs work better
Breathing function as measured by pulmonary function tests improved for patients in the long-term extension study compared to baseline.
FVC, volume of air that can forcibly be exhaled from the lungs after taking the deepest breath possible
FEV1, maximum volume of air that can be forcibly blown out in 1 second.
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.
Serious and severe reactions were associated with VIMIZIM® (elosulfase alfa), including hypersensitivity reactions and life-threatening allergic reactions (anaphylaxis).
In clinical trials, 18.7% of patients treated with VIMIZIM experienced hypersensitivity reactions and 7.7% experienced anaphylaxis.
Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
See the side effects of VIMIZIM
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.