Two of Alexion’s rare-disease drugs get the thumbs up from the EU

01/07/2015 - 2 minutes

The European Medicines Agency (EMA) is about to approve a couple of rare diseases drugs developed by Alexion. The two enzymes, Strensiq and Kanuma, received  positive feedback from the EMA’s Committee for Medicinal Products for Human Use, which normally leads to the approval of the drug.

The Committee for Medicinal Products for Human Use has recommended the marketing authorization of Alexion’s Strensiq and Kanuma. Strensiq, asfotase alfa, is indicated in patients with pediatric-onset hypophosphatasia to treat the bone deformities of the disease. On the other side, Kanuma, sebelipase alfa, is indicated for lysosomal acid lipase deficiency. Two rare disorders with no therapies approved for their treatment.

Whilst Strensiq is an in-house product, Kanuma was obtained thanks to Alexion’s purchase of Synageva. The rare-diseases expert was acquired for a total amount of €7.6Bn, and the candidate was integrated in Alexion’s rare diseases pipeline.

Synageva’s buying is a great milestone for Alexion, that now owns one of the most robust rare disease pipelines in the biotech industry across a wide range of therapeutic modalities.

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