What is Aldurazyme?
Aldurazyme is a solution for infusion (phleboclysis) that contains the active substance laronidase.
What is Aldurazyme used for?
Aldurazyme is indicated in patients with a confirmed diagnosis of mucopolysaccharidosis I (MPS-I; α-L-iduronidase deficiency), for the treatment of non-neurological manifestations (not connected with the brain or nerves) of the disease. MPS I is a rare inherited disease in which the level of α-L-iduronidase enzyme activity is much lower than normal. Consequently, there is no degradation of certain substances (glycosaminoglycans), which accumulate in most of the organs of the body and damage them. The non-neurological manifestations of MPS I may be liver enlargement, joint stiffness resulting in difficulty moving, decreased lung volume, heart disease and eye diseases. Because the number of patients with MPS is low, the disease is considered "rare" and Aldurazyme was given the status of an orphan medicine (a medicine used for rare diseases) on February 14, 2001. The medicine can only be obtained with a prescription. medical.
How is Aldurazyme used?
Treatment with Aldurazyme should be under the supervision of a doctor experienced in the management of patients with MPS I or other inherited metabolic diseases. Aldurazyme should be administered in hospitals or clinics where resuscitation equipment is available and it is possible that other medications should be given before the infusion to prevent allergic reactions. Aldurazyme is given once a week as an intravenous infusion and is indicated for long-term therapy.
How does Aldurazyme work?
The active substance in Aldurazyme, laronidase, is a copy of the human enzyme α-L-iduronidase. it is produced by a method known as "recombinant DNA technology": the enzyme is produced by a cell in which a gene (DNA) has been introduced which makes it capable of producing laronidase, which is used as "enzyme replacement therapy", ie it replaces the missing enzyme in patients with MPS I. The symptoms of MPS I are thus controlled, with the consequent improvement in the quality of life of the patients.
How has Aldurazyme been studied?
Aldurazyme was compared with a placebo (a dummy treatment) in 45 patients aged 5 years and with confirmed diagnosis of mucopolysaccharidosis I (MPS-I). The main measure of effectiveness was the forced vital capacity (CVF, a measure of pulmonary efficacy) and the distance patients could walk for six minutes. These values were measured before and after 26 weeks of therapy. After that the study continued for a maximum period of four years and all patients were treated with Aldurazyme. Aldurazyme has been studied in 20 children under the age of five, who were given Aldurazyme for a year. The study focused mainly on the safety of the medicine, but it also measured its ability to reduce GAG levels in urine and the size of the liver.
What benefit has Aldurazyme shown during the studies?
The study showed that Aldurazyme improved both the forced vital capacity (CVF) and the patient's ability to walk after 26 weeks and that the effect was maintained for up to four years. In children under the age of five, Aldurazyme reduced urine glycosaminoglycan levels by about 60% and half of the children treated had a normal-sized liver at the end of the study.
What is the risk associated with Aldurazyme?
Most of the side effects seen with Aldurazyme are reactions caused by the infusion rather than the medicine itself. Some of them are serious, but the number of side effects tends to decrease over time. The most common side effects with Aldurazyme seen in patients over the age of five (more than one in ten patients) are headache, nausea, abdominal pain (stomach ache), rash, arthropathy (joint degeneration), arthralgia (joint pain), back pain, extremity pain (hands and feet), flushing, pyrexia (fever) and infusion site reactions. In patients under the age of five, the most common side effects are increased blood pressure, decreased oxygen saturation (a measure of the effectiveness of lung function), tachycardia (increased heart rate), pyrexia and chills. For the full list of all side effects reported with Aldurazyme, see the Package Leaflet. Patients receiving Aldurazyme often develop antibodies (proteins produced in response to the medicine), but the effect that these antibodies can have on the safety and efficacy of the drug is not yet fully known. Aldurazyme should not be given to subjects who may have serious allergic reactions (such as an anaphylactic reaction) to laronidase or other excipients.
Why has Aldurazyme been approved?
The Committee for Medicinal Products for Human Use (CHMP) decided that Aldurazyme effectively controls the symptoms of MPS I. The committee decided that the benefits of Aldurazyme outweigh the risks for long-term enzyme replacement therapy in patients with a confirmed diagnosis of MPS I. The Committee recommended that it be given marketing authorization. Aldurazyme has been authorized "in exceptional circumstances", which means that since it is used to treat a rare disease, more detailed information on the medicine could not be obtained. The European Medicines Agency reviews the new information available every year and, if necessary, updates this summary.
What information is still awaited for Aldurazyme?
The company that makes Aldurazyme will monitor the patients taking the drug by observing the infusion reactions and the development of antibodies.
Other information:
On 10 June 2003, the European Commission granted a marketing authorization for Aldurazyme to Genzyme Europe BV, valid throughout the European Union. The marketing authorization was renewed on 10 June 2008. For a summary of the opinion of the Committee for Orphan Medicinal Products for Aldurazyme, click here. For the full EPAR of Aldurazyme, click here .
Last update of this summary: 06-2009.
