First-In-Class Enzyme Replacement Therapy for Hypophosphatasia Under Review

Alexion announced that the FDA has accepted for Priority Review the Biologics License Application (BLA) for asfotase alfa in the treatment of patients with infantile-and juvenile-onset hypophosphatasia (HPP). 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 classified by the age of the patient at the onset of symptoms of the disease, with infantile- and juvenile-onset HPP defined as manifestation of the first symptom prior to 18 years of age.

The BLA submission for asfotase alfa is supported by data from 71 treated patients with HPP enrolled in three prospective studies and their extensions, as well as two retrospective natural history studies.

Asfotase alfa is an investigational, first-in-class enzyme replacement therapy designed to address the underlying cause of HPP. It aims to restore the genetically defective metabolic process, thereby preventing or reversing the severe and potentially life-threatening complications of life-long dysregulated mineral metabolism.

In May 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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