VIMIZIM® (elosulfase alfa) works to help with deficient enzyme activity

VIMIZIM® (elosulfase alfa) works at a cellular level to help with deficient enzyme activity

How VIMIZIM works

VIMIZIM 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.

5 Patients showing range of presentation of Morquio A

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.

 
 
Outline of a patient's body showing GAG buildup in various areas of the body

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

Close-up of cell showing increased GALNS enzyme in the lysosomes

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

Outline of patient's body showing increased GALNS

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

Endurance with VIMIZIM vs placebo at 24 weeks

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 BioMarin Pharmaceutical Inc. at 1-866-906-6100, or FDA at 1-800-FDA-1088 or go to www.fda.gov/medwatch.

Learn more about the possible side effects of VIMIZIM

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.

Learn more about the possible side effects of VIMIZIM

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

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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 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, as early as 30 minutes from the start and up to 3 hours after infusion, and as late into treatment as the 47th infusion. Hypersensitivity reactions have been observed as early as 30 minutes from the start of infusion but as late as 6 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 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 at 1-866-906-6100 and the FDA by visiting www.fda.gov/medwatch or calling 1-800-FDA-1088.

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

Please see accompanying full Prescribing Information, including important warning.

US-VIM-00085 0323