25 June 2014

Vimizim for the treatment rare disease Morquio A syndrome

The drug Elosulfase Alfa with the brand name Vimizim was approved by U.S. FDA on  February 2014, for the treatment of Morquio A Syndrome (MPS IVA). Morquio A is a rare inherited disease which is also known as mucopolysaccharidosis IV A. Mucopolysaccharidosis refers to a class to diseases in which are related to the metabolic disorders in Mucopolysaccharides and could be of different subtypes.

Morquio A is a rare inherited disease which  affects major organ systems in the body. The disease is characterized by mucopolysaccharidosis, a type of lysosomal storage disorder. People born with Morquio A can't break down glycosaminoglycans (GAGs) molecules because their bodies couldn’t make enough of the enzyme N-acetylgalactosamine-6 sulfatase (GALNS). This enzyme breaks down or recycles materials the body can't use. When the body couldn'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. Physical growth slows generally around the age of 18 months, and stops completely by the age of 8.

The symptoms of this disease are abnormal development of bones, bell-shaped chest, coarse facial features, hyper mobile joints, knock- knees, widely spaced teeth. 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 gets 2 or 3 years old, enough GAGs have built up in the child’s body to start causing noticeable problems. Even though Morquio A is serious disease but the disease can affect people differently. Children with the more severe form of Morquio A syndrome may not live beyond their twenties or thirties. With proper treatment, people with Morquio A could  live a long time  in some cases, just as long as normal people.

The safety and efficacy of Vimizim were estimated by establishing a clinical trial in which 176 subjects with Morquio A Syndrome ranging from age group 5 to 57 years were included. In this clinical trial the drug was administered by intravenous infusion and was supplied as a sterile, nonpyrogenic, colorless to pale yellow, clear to slightly opalescent solution which was diluted with 0.9% Sodium Chloride for injection. The subjects treated with Vimizim showed an improvement in 6 minutes walk then compared to the subjects administered with Placebo. Overall the patients treated with Vimizim in the trail walked 22.5 meters in 6 minutes compared to the patients who received placebo.

But before Vimizim treatment a patient has to be pretreated with antihistamines (with or without antipyretics) 30-60 minutes before initiation of infusion. The drug works on a cellular level to restore cell function. Vimizim improved endurance as measured by the 6-minute walk test (6MWT).This drug is a prescription medicine. Before starting treatment with Vimizim, it is important to discuss the patient’s medical history with the doctor. Female patients should  discuss with their doctor if they  are pregnant or  planning to become pregnant, or  a nursing mother. The most common side effects included fever, vomiting, headache, nausea, abdominal pain, chills, and fatigue. Anaphylaxis could  occur during any drug infusion and could occur 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 the drug Vimizim treatment, including life-threatening allergic reactions (anaphylaxis), hives, swelling, cough, shortness of breath, and flushing.

This drug the only enzyme replacement therapy (ERT) to address the cause of Morquio A (MPS IVA). Thus it would of great benefit to treat the patients, although proper medical history as well different medical checkups should be done and proper medical advice should be taken before starting  treatment with Vimizim.

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