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Alexion scores double EU approval

Published on 01/09/15 at 10:34am
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Two treatments developed by ultra-rare disease specialists Alexion Pharmaceuticals are now approved for use in Europe.

The European Commission approved the two long-term enzyme replacement therapies – Kanuma (sebelipase alfa), which is a treatment for patients with lysosomal acid lipase deficiency (LAL-D), and Strensiq (asfotase alfa), for children with paediatric-onset hypophosphatasia (HPP) to treat the bone problems caused by the disease.

Both Kanuma and Strensiq are the first therapies approved in the European Union for the treatment of patients with these ultra-rare disorders, which are defined as those that affect fewer than 20 in a million people in the general population.

Alexion says it expects to begin providing both drugs to patients in Germany in October and “is now commencing reimbursement processes with healthcare authorities in each of the major European countries.”

LAL-D is a genetic, chronic and progressive metabolic disease in which infants, children and adults suffer multi-organ damage and premature death. Patients with LAL-D often experience a rapid onset of life-threatening disease manifestations, and similar to other liver diseases, many patients may be asymptomatic until they experience a severe consequence of the disease. LAL-D is caused by genetic mutations that result in a marked decrease or loss in LAL enzyme activity in the lysosomes across multiple body tissues, leading to the chronic build-up of cholesteryl esters and triglycerides in the liver, blood vessel walls and other tissues.

HPP is a genetic and progressive metabolic disease which affects multiple systems of the body, leading to debilitating or life-threatening complications. HPP can lead to deformity of bones and other skeletal abnormalities, as well as complications such as muscle weakness, seizures, pain, and respiratory failure leading to premature death in infants.

David Hallal, chief executive of Alexion, says: “We are grateful to the investigators, patients, and their families who participated in the clinical trials that made this approval possible and we are now commencing reimbursement processes with healthcare authorities throughout Europe to ensure that patients with [these conditions] have access to a life-transforming treatment, as quickly as possible.”

Alexion has previously developed and brought a treatment for an extremely rare condition, atypical haemolytic uraemic syndrome (aHUS), to market in the UK when it earned NICE approval for Soliris (eculizumab). In May Alexion spent $8.4 billion to acquire the US orphan drug developer Synageva.

Lilian Anekwe

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