Data from Pharmawand - Curated by EPG Health - Date added 02 September 2015

Alexion Pharmaceuticals, Inc. announced that the European Commission has approved Kanuma (sebelipase alfa) for long-term enzyme replacement therapy in patients of all ages with lysosomal acid lipase deficiency (LAL-D). Kanuma is the first approved treatment in the European Union for patients with LAL-D, a genetic and progressive ultra-rare metabolic disease in which patients suffer multi-organ damage and premature death. Alexion expects to begin serving patients in Germany in October and is now commencing reimbursement processes with healthcare authorities in each of the major European countries.

The approval of Kanuma in the EU was based on data from two clinical studies and a supporting open-label extension study comprising infant, paediatric, and adult patients with LAL-D. Study results showed significant benefit in terms of survival (67%, or 6 out of 9) in patients with the infant form of LAL-D beyond 12 months, compared with 0 out of 21 patients in an untreated historical cohort. Infant patients treated with Kanuma also had improvements in liver parameters, including ALT and AST, as well as weight gain within the first several weeks of treatment. In paediatric and adult patients with LAL-D, treatment with Kanuma resulted in normalisation of ALT, reduction in liver fat content and other markers of liver injury compared to placebo, as well as significant improvements in lipid accumulation as measured by LDL-C and HDL-C. In patients who received Kanuma during the double-blind period and subsequently entered the open-label extension period, reductions in ALT levels were maintained and further improvements were seen in LDL-C and HDL-C.

Comments

You will need to login, to leave a comment.

epgonline.org is not monitored for collection of adverse event reports. Any adverse events should be reported to your national reporting agency and/or the manufacturer.

Learning Zones

An epgonline.org Learning Zone (LZ) is an area of the site dedicated to providing detailed self-directed medical education about a disease, condition or procedure.

Hereditary ATTR amyloidosis

Hereditary ATTR amyloidosis

Explore the pathophysiology, epidemiology and multi-system symptoms associated with hereditary ATTR amyloidosis, as well as how to achieve an early and accurate diagnosis.

Sleep Apnea

Sleep Apnea

Sleep apnea is a chronic condition characterised by recurrent interruptions in breathing throughout the sleep cycle. Learn about the two main types of sleep apnea and hear from experts as they present the latest research at the Sleep and Breathing conference (Marseille, 2019).

Oral Anticoagulation Reversal

Oral Anticoagulation Reversal

Experts discuss the use of non-vitamin K oral anticoagulants in the treatment and prevention of stroke, deep vein thrombosis and pulmonary embolism in atrial fibrillation patients.

Load more

Related Content