Alexion Pharmaceuticals has announced that the European Medicines Agency (EMA) has validated and granted accelerated assessment for its marketing authorisation application for asfotase alfa to treat hypophosphatasia.
EMA’s acceptance of the marketing authorisation application marks the beginning of the review process in the EU for asfotase alfa, an investigational, first-in-class targeted enzyme replacement therapy.
The company’s submission of the application is based on positive data from three pivotal prospective studies and their extensions, as well as a retrospective natural history study in infants.
These studies included 68 paediatric-onset hypophosphatasia patients who range from newborns to 66 years of age.
Alexion Pharmaceuticals CEO Leonard Bell said: "Hypophosphatasia is a devastating disease for patients and their families due to progressive deterioration of bones and muscle weakness, which can result in impaired respiratory function, severe disability and death.
"If approved, asfotase alfa would be the first therapy for patients with this life-threatening disorder."
The company initiated the rolling submission of a biologics licenxe application for asfotase alfa with the US Food and Drug Administration (FDA) in April for treatment of hypophosphatasia. Asfotase alfa was granted breakthrough therapy designation by the FDA in 2013.
Hypophosphatasia is a genetic, chronic and progressive ultra-rare metabolic disease characterised by defective bone mineralisation that can lead to destruction and deformity of bones, profound muscle weakness, seizures, respiratory failure and premature death.
Asfotase alfa is developed to address the underlying cause of hypophosphatasia by normalizing the genetically defective metabolic process, and preventing or reversing the severe and potentially life-threatening complications of life-long dysregulated mineral metabolism.
