Naglazyme

Описание товара

About Naglazyme

Naglazyme is a solution for infusion (drip into a vein) that contains the active substance galsulfase (1 mg/ml).

What it is used for

Naglazyme is used to treat patients who have mucopolysaccharidosis VI (MPS VI or Maroteaux-Lamy syndrome). This disease is caused by the lack of an enzyme called N-acetylgalactosamine 4-sulfatase, which is needed to break down substances in the body called glycosaminoglycans (GAGs). If the enzyme is not present, GAGs cannot be broken down and they build up in the cells. This causes the signs of the disease, the most noticeable being a short body, a large head and difficulty moving about. The disease is usually diagnosed in infants between one and five years of age. Because the number of patients with MPS VI is low, the disease is considered ‘rare’, and Naglazyme was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 14 February 2001. The medicine can only be obtained with a prescription.

How it is used

Naglazyme treatment should be supervised by a doctor who has experience in the management of patients with MPS VI or similar diseases. It should be given where resuscitation equipment is available in case of a medical emergency. Naglazyme is given as a four-hour infusion once a week. The recommended dose is 1 mg per kilogram bodyweight. Before each infusion, patients should be given an antihistamine to reduce the risk of an allergic reaction. Patients may also be given a medicine to prevent fever.

How it works

Naglazyme is an enzyme replacement therapy. Enzyme replacement therapy provides patients with the enzyme they are lacking. The active substance in Naglazyme, galsulfase, is a copy of the human enzyme N-acetylgalactosamine 4-sulfatase. Naglazyme helps to break down the GAGs and stop them building up in the cells. This can improve the symptoms of MPS VI, including the ability of patients to move about. Galsulfase is produced by a method known as ‘recombinant DNA technology’: it is made by a cell that has received a gene (DNA), which makes it able to produce the enzyme.

Active substance: galsulfase

Source: EMA EPAR