If ever a drug development effort called out for priority review and approval, this was it. A U.S. pharmaceutical company was developing a treatment for hypophosphatasia, a progressive, ultra-rare metabolic disease for which only supportive therapy was available. Asfotase alfa, an innovative enzyme replacement drug, was the first real hope for those diagnosed with HPP, a disease that attacks bones and multiple systems of the body with devastating consequences.
The Food and Drug Administration granted the compound Breakthrough Therapy designation and issued the developer a Rare Pediatric Disease Priority Review Voucher, which confers priority review to a subsequent drug application that would not otherwise qualify for priority status. The voucher program encourages development of new drugs and biologics to prevent or treat rare pediatric diseases. Meanwhile, the European Medicines Agency conferred orphan status and provided scientific advice through its Committee for Medicinal Products for Human Use.
The expedited development and subsequent approval of the drug – on the market now in the United States, European Union, Japan, and Canada – reflects a large and promising trend in the battle against rare diseases. Concerted effort by governments, regulators, and industry is expanding development of new therapies, opening doors and giving fresh hope to the hundreds of millions who suffer from the more than 7,000 known rare diseases.