Vimizim® (elosulfase alfa) for Morquio A Syndrome (MPS IVA)
Vimizim® (elosulfase alfa) is the only enzyme replacement therapy (ERT) to address the cause of Morquio A (MPS IVA). People born with Morquio A have a gene that does not work correctly. This gene results in missing or low levels of the N-acetylgalactosamine-6 sulfatase (GALNS) enzyme that breaks down certain complex carbohydrates known as glycosaminoglycans (GAGs). Without sufficient quantities of GALNS, the normal breakdown of GAG is incomplete or blocked and it accumulates in the lysosomes of the cell.
This accumulation disrupts the cell’s normal functioning and gives rise to the clinical manifestations. Weekly infusions of Vimizim can help break down some of the GAGs in the body that cause the signs and symptoms of Morquio A. Vimizim works on a cellular level to restore cell function. Vimizim can help improve endurance as measured by the 6-minute walk test (6MWT).
- Approved in the United States, European Union, Canada, Japan, Australia, Mexico, and Brazil
- Manufactured and commercialized by BioMarin
- Designated as an orphan drug in the United States and the European Union
The U.S. Food and Drug Administration approved Vimizim® in February 2014, the first and only therapy designed to address Morquio A syndrome at the cellular level. In April, the European Commission granted marketing authorization to Vimizim®, the first drug ever approved for Morquio A syndrome with orphan drug status. The European Commission conferred orphan drug status on Vimizim®, which provides ten years of market exclusivity. In July 2014, Health Canada approved Vimizim®, the only pharmaceutical treatment option available in Canada for children and adults living with this severely debilitating, progressive and life-limiting disorder. BioMarin has also won approval for Vimizim® in Brazil, Australia, Canada, Mexico, and Japan.
Vimizim® is manufactured by BioMarin in the United States and is commercialized by BioMarin in the United States, European Union, and Canada.
About Morquio A
Morquio A is a rare inherited disease that affects major organ systems in the body. The disease is a form of mucopolysaccharidosis, which is a type of lysosomal storage disorder. People born with Morquio A can’t break down glycosaminoglycans (GAGs) molecules because their bodies don’t make enough of an enzyme, or protein, called N-acetylgalactosamine-6 sulfatase (GALNS). This enzyme breaks down or recycles materials the body can’t use. When the body doesn’t produce enough of the enzyme, GAGs build up in tissues, bones, and major organs. GAGs cause serious problems, including heart disease, skeletal abnormalities, vision and hearing loss, difficulty breathing, and early death. Even though Morquio A is serious, it’s important to remember that the disease can affect people differently.
People with Morquio A can live a long time—in some cases, just as long as people who don’t have Morquio A.
Signs and Symptoms
When GAGs build up in the bodies of people with Morquio A, the results can potentially be severe. People with Morquio A have physical problems and organ complications that can lead to difficulties performing daily activities, and reduced endurance may cause them to need walking aids or wheelchairs. The signs and symptoms of Morquio A are not usually easy to see at birth, however, by the time the child is 2 or 3 years old, enough GAGs have built up in the child’s body to start causing noticeable problems. The signs and symptoms of Morquio A can affect many different parts of the body and frequently include heart, lung, visual, hearing, and dental complications, in addition to the skeletal problems that are typically present in people with Morquio A syndrome.
- Skeletal deformities
- Short trunk
- Weakness in the neck
- Spinal cord compression
- Knock knees
- Overly flexible joints
- Abnormal walk
- Spinal problems
- Chest deformities
- Breathing problems
- Heart valve problems
- Muscle weakness
- Eye problems
- Hearing loss
- Dental problems
- Enlarged liver
- Enlarged spleen
- Limited energy
Getting a diagnosis of Morquio A is an important first step toward getting treatment.
Vimizim 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, taking any medication, or 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.
Please see full Prescribing Information by going to vimizim.com, including important warning.
For additional information about MPS IVA, please visit the following websites and others listed in the Patient/Physician Resource Library: