FDA Approves Alexion’s Ultra-Rare Drug

The US Food and Alexion Pharmaceuticals, Inc. announced today that the US Food and Drug Administration (FDA) has approved Strensiq (asfotase alfa) for the treatment of patients with perinatal-, infantile- and juvenile-onset hypophosphatasia (HPP). Strensiq, an enzyme replacement therapy (ERT), is approved in the US for the treatment of patients with HPP, a genetic, chronic, and progressive ultra-rare metabolic disease in which patients experience devastating effects on multiple systems of the body.

HPP is characterized by low alkaline phosphatase (ALP) activity and defective bone mineralization that can lead to deformity of bones and other skeletal abnormalities as well as systemic complications such as profound muscle weakness, seizures, pain, and respiratory failure leading to premature death in infants. HPP is an ultra-rare disease, which is defined as a disease that affects fewer than 20 patients per one million in the general population.

Alexion will now begin serving patients with HPP in the U.S., with Strensiq becoming available commercially by October 27, 2015. The FDA approved Strensiq under priority review, and had granted breakthrough therapy designation for Strensiq. With this approval, the FDA also issued a rare pediatric disease priority review voucher, which confers priority review to a subsequent drug application that would not otherwise qualify for priority review. The rare pediatric disease review voucher program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases. Strensiq is also approved in the European Union, Japan, and Canada.

Source: Alexion Pharmaceuticals

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