Kanuma - Sebelipase alfa

What is Kanuma - Sebelipase alfa used for?

Kanuma is a medicine used to treat patients of all ages with lysosomal acid lipase deficiency, an inherited disorder caused by the lack of an enzyme called lysosomal acid lipase, which is necessary to metabolize fats in cells. When this enzyme is absent or is present only in very low amounts, fats accumulate in the body's cells, causing symptoms such as growth deficiency and liver damage.

Because the number of patients with lysosomal acid lipase deficiency is low, the disease is considered 'rare' and Kanuma was designated an 'orphan medicine' (a medicine used in rare diseases) on 17 December 2010.

Kanuma contains the active substance sebelipase alfa.

How is Kanuma used - Sebelipase alfa?

Treatment with Kanuma should be followed by a doctor experienced in treating lysosomal acid lipase deficiency, other metabolic disorders or liver disease. Treatment should be administered by a suitably trained healthcare professional who can handle medical emergencies (including severe allergies). The medicine can only be obtained with a prescription.

Kanuma is available as a concentrate for solution for infusion (drip into a vein). The recommended dose is 1 mg per kilogram of body weight, to be given once every two weeks. The infusion should last about 1-2 hours.

In patients under six months of age who present with the rapidly progressing disease, the dose of 1 mg / kg of body weight is administered once a week instead of every two weeks; in these subjects the dose can be increased up to 3 mg / kg of body weight once a week, depending on the response to treatment.

Treatment with Kanuma should be started as soon as possible after diagnosis and continued in the long term.

How does Kanuma - Sebelipase alfa work?

The active substance in Kanuma, sebelipase alfa, is a copy of the enzyme that is missing in patients with lysosomal acid lipase deficiency. Sebelipase alfa replaces the missing enzyme, contributing to the metabolism of fat and stopping its accumulation in the body's cells.

What benefit has Kanuma - Sebelipase alfa shown during the studies?

Kanuma has been studied in two main studies conducted in patients with lysosomal acid lipase deficiency. The first study involved nine small children with growth failure or other signs of rapid disease progression in the first six months of life. The study showed that six out of nine children treated with Kanuma reached the age of one. Growth improvements have also been observed in all six surviving children.

The second study, which involved 66 patients (children and adults), compared Kanuma with placebo (a dummy treatment). The main measure of effectiveness was the percentage of patients in whom normal levels of a liver enzyme called ALT were observed after five months of treatment. Elevated levels of ALT enzymes are a sign of liver damage. In this study, normal levels of ALT enzymes were found in 31% (11 of 36 patients) of subjects treated with Kanuma compared to 7% (2 of 30) of subjects given placebo

What is the risk associated with Kanuma - Sebelipase alfa?

The most common side effects of Kanuma (which may affect about 3 out of 100 patients) are the signs and symptoms of severe allergic reactions, including chest tightness, red eyes, swelling of the eyelids, difficulty breathing, hives, wheals, flushing heat, nasal discharge, tachycardia and respiratory distress. Development of antibodies against the medicine, especially in young children, has also been reported. In this case, Kanuma may not act effectively. For the full list of all side effects reported with Kanuma, see the package leaflet.

Kanuma should not be used in patients who have experienced life-threatening allergic reactions to the active ingredient, which have recurred with the resumption of treatment after an interruption. It must also not be given to patients with a potentially lethal allergy to eggs or any of the ingredients of Kanuma.

Why has Kanuma - Sebelipase alfa been approved?

The Agency's Committee for Medicinal Products for Human Use (CHMP) decided that Kanuma's benefits are greater than its risks and recommended that it be approved for use in the EU. The committee took into account the absence of effective therapies for lysosomal acid lipase deficiency and the high mortality rate in young children with rapidly progressing disease. The CHMP considered that Kanuma induced significant improvements in survival in young children and proved effective in alleviating the symptoms of the disease in individuals of all ages. As for safety, no major problems were identified, while serious side effects were rare or manageable. However, additional data on the long-term benefits and safety of the medicine are needed.

What measures are being taken to ensure the safe and effective use of Kanuma - Sebelipase alfa?

A risk management plan has been developed to ensure that Kanuma is used as safely as possible. Based on this plan, safety information has been included in the summary of product characteristics and the package leaflet for Kanuma, including the appropriate precautions to be followed by healthcare professionals and patients.

Furthermore, the company that markets Kanuma is carrying out a study in young children with rapidly progressing illness and will establish a registry of patients of all ages to learn more about the benefits and safety of Kanuma in the long term, with particular regard to the risk of allergic reactions and the development of antibodies against the medicine. The company will also provide informative material to all doctors who could prescribe Kanuma, inviting them to register patients in the register and indicating how to monitor patients for the development of antibodies and how to manage those with severe allergic reactions.