WARNING: Life-threatening allergic reactions have occurred in some patients during VIMIZIM® (elosulfase alfa) infusions and up to 3 hours after infusion. Patients with acute respiratory illness may be at increased risk and require additional monitoring.

In a 6-month clinical trial, people who took VIMIZIM improved their endurance as measured by the 6-minute walk test (6MWT).

In a 6-month clinical trial, people who took VIMIZIM<sup>®</sup> (elosulfase alfa) improved their endurance.” width=”760″ height=”431″ class=”alignnone size-full wp-image-2726″ /></p>
<p class=aA baseline is a measurement of a person’s performance before they begin treatment in a clinical trial. In order to participate in the clinical trial for VIMIZIM, people had to be able to walk between 30 meters and 325 meters.

bA placebo is a substance that does not contain any medicine.

This graph shows that people who took VIMIZIM® (elosulfase alfa) once a week for 24 weeks improved their endurance over time. At the end of 24 weeks, these people were able to walk 23.9% farther than they could at the start of the trial.

The trial also included a group of people who were given VIMIZIM 2 mg/kg every other week. Their results did not show much improvement and were similar to the people who took placebo.

Why use the 6MWT?

The “International Guidelines for the Management and Treatment of Morquio A Syndrome” establish that the 6MWT is the most effective measure of endurance. The test measures how far you can walk in 6 minutes. More importantly, it measures your endurance—or how far you can push yourself physically. Endurance measures how well your whole body performs—from heart and lungs to bones and muscles. When your endurance improves, your body’s systems are working better together.

In clinical studies, VIMIZIM helped most people with their endurance, as measured by the 6MWT—no matter how serious their Morquio A symptoms were. And because Morquio A affects every person in different ways, the results you’ll see from VIMIZIM will be specific to you.

See how VIMIZIM changed Annabelle’s life.

Need a geneticist? Find one with experience in Morquio A near you.

What should I know before taking VIMIZIM?


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 FDA at 1-800-FDA-1088.

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


BioMarin Pharmaceutical Inc.

Novato, CA 94949

BioMarin RareConnections™: 1-866-906-6100