Cheshire-based Alexion Pharmaceuticals has received U.S. Food and Drug Administration approval for Strensiq. It’s used in the treatment of patients with perinatal-, infantile- and juvenile-onset hypophosphatasia.
Strensiq, according to an Alexion statement, is an innovative enzyme replacement therapy. It is the first therapy approved in the U.S. for the treatment of patients with hypophosphatasia, a genetic and progressive ultra-rare metabolic disease in which patients experience devastating effects on multiple systems of the body, leading to debilitating or life-threatening complications.
“The FDA approval of Strensiq brings a highly innovative treatment to patients who, until now, have had no effective therapy to treat this ultra-rare genetic metabolic disease that causes premature death in infants and devastating consequences in those who survive,” said David Hallal, Alexion CEO.
Hypophosphatasia is characterized by low alkaline phosphatase activity and defective bone mineralization that can lead to deformity of bones and other skeletal abnormalities, as well as systemic complications such as profound muscle weakness, seizures, pain, and respiratory failure leading to premature death in infants. It is classified as an ultra-rare disease in that it affects fewer than 20 patients per one million in the general population.
The drug becomes available commercially tomorrow, Oct.27. Strensiq is also approved in the European Union, Japan, and Canada.
