EMA Accepts Alexion's MAA For Hypophosphatasia Drug

The European Medicines Agency (EMA) has accepted Alexion Pharmaceuticals’ Marketing Authorization Application (MAA) for its investigational drug asfotase alfa for the treatment of hypophosphatasia (HPP). The EMA has also granted accelerated assessment for the company’s application.

Asfotase alfa is a first-in-class enzyme replacement therapy designed to target the underlying cause of the rare disease HPP. The drug works by normalizing genetically defective metabolic processes and preventing or reversing the serious, potentially life-threatening complications of lifelong faulty mineral metabolism. Asfotase alfa was granted Breakthrough Therapy by the U.S. Food and Drug Administration (FDA) last year.

Hypophosphatasia is an ultra rare, chronic, progressive, genetic disease characterized by defective bone mineralization. HPP can cause destruction and deformity of the bones and affect muscle functions. Symptoms also include seizures, respiratory failure, and even premature death. The disease is caused by the genetic encoding of the tissue non-specific alkaline phosphatase (TNSALP) enzyme. HPP’s genetic deficiency affects people of all ages.

Leonard Bell, CEO of Alexion, said, “HPP is a devastating disease for patients and their families... If approved, asfotase alfa would be the first therapy for patients with this life-threatening disorder.”

The European submission for asfotase alfa is supported by positive results from three pivotal studies involving 68 patients with pediatric-onset HPP. Alexion started the rolling submission for asfotase alfa’s Biologics License Application (BLA) with the FDA in April.

Last month the company announced the launch of a research collaboration in complex and rare diseases with partner Cincinnati Children's Hospital Medical Center. As part of the agreement, Alexion gains the option to fund chosen research programs through its Rare Disease Innovation Fund and to sign a licensing agreement for these programs.

The company also announced the appointments of David R. Brennan, M. Michele Burns and Christopher J. Coughlin to the Alexion's Board of Directors earlier this July.