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Alexion Pharmaceuticals, Inc.
announced today completion of the
rolling submission of a Biologics License Application (BLA) to the U.S. Food
and Drug Administration (FDA) for asfotase alfa, an investigational,
first-in-class enzyme replacement therapy for the treatment of
hypophosphatasia (HPP). 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.
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