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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.For more click here