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news.Alexion Pharmaceuticals announced completion of the rolling submission of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for asfotase alfa, an investigational, first-in-class enzyme replacement therapy for the treatment of hypophosphatasia (HPP).
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HPP is a genetic, chronic and progressive ultra-rare metabolic disease that can lead to destruction and deformity of bones, profound muscle weakness, seizures, respiratory failure and premature death.1-5
"Completion of the rolling BLA is another step forward in our goal to bring this highly innovative potential treatment to patients with HPP who currently have no approved treatment options," said Leonard Bell, M.D., Chairman and Chief Executive Officer of Alexion.
"Our goal in all territories is to continue to work with regulatory authorities to obtain marketing authorizations for asfotase alfa and to bring this important therapy to patients with HPP as quickly as possible."
The BLA submission includes data from 71 treated patients with HPP enrolled in three prospective studies and their extensions, as well as a retrospective natural history study in infants with HPP and a separate retrospective natural history study in juveniles with HPP.
In 2013, the FDA granted Breakthrough Therapy designation for asfotase alfa and in April 2014, Alexion initiated the rolling submission of the BLA.
HPP is a genetic, chronic and progressive ultra-rare metabolic disease characterized by defective bone mineralization that can lead to destruction and deformity of bones, profound muscle weakness, seizures, respiratory failure and premature death.
HPP is caused by mutations in the gene encoding an enzyme known as tissue non-specific alkaline phosphatase (TNSALP).1,2 The genetic deficiency in HPP can affect people of all ages. HPP is classified by the age of the patient at the onset of symptoms of the disease, and infantile- and juvenile-onset HPP is defined as manifestation of the first symptom prior to 18 years of age.