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Alexion Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration (FDA) has approved Strensiq (asfotase alfa) for the treatment of patients with perinatal-, infantile- and juvenile-onset hypophosphatasia (HPP).
HPP is a rare, genetic, progressive, metabolic disease in which patients experience devastating effects on multiple systems of the body, leading to severe disability and life-threatening complications. It is characterized by defective bone mineralization that can lead to rickets and softening of the bones that result in skeletal abnormalities. It can also cause complications such as profound muscle weakness with loss of mobility, seizures, pain, respiratory failure and premature death. Severe forms of HPP affect an estimated one in 100,000 newborns, but milder cases, such as those that appear in childhood or adulthood, may occur more frequently.
Strensiq is administered via injection three or six times per week. Strensiq, an innovative enzyme replacement therapy (ERT), is the first therapy approved in the U.S. 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, leading to debilitating or life-threatening complications.
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