The European Commission (EC) has granted marketing authorization for Strensiq, which treats the bone manifestation of pediatric-onset hypophosphatasia (HPP).
Alexion said HPP is a genetic and progressive metabolic disease in which patients experience devastating effects on several systems of the body, resulting in debilitating or life-threatening complications.
Alexion Pharmaceuticals CEO David Hallal said: "As the first approved treatment for pediatric-onset HPP in Europe, Strensiq is an innovative therapy for patients suffering from this devastating and life-threatening ultra-rare disease.
"We are pleased that the EU label will allow any patient who had symptoms of HPP prior to the age of 18 to be eligible for treatment,"
The commission also gave authorisation for the company’s Kanuma, which treats people with lysosomal acid lipase deficiency.
LAL-D is a genetic, chronic and progressive metabolic disease that leads to multi-organ damage and premature death.
Hallal said: "In the absence of any effective therapy, patients with LAL-D face devastating morbidities including liver failure and premature mortality.
"We are grateful to the investigators, patients, and their families who participated in the clinical trials that made this approval possible and we are now commencing reimbursement processes with healthcare authorities throughout Europe to ensure that patients with LAL-D have access to Kanuma, a life-transforming treatment, as quickly as possible."
Both HPP and LAL-D are defined as ultra-rare diseases occurring in fewer than 20 patients per one million in the general population.
The EC approval of Strensiq and Kanuma applies to all 28 European Union member states as well as Iceland, Norway, and Lichtenstein.
Alexion expects to start serving patients in Germany in October and is beginning reimbursement processes with healthcare authorities in major European countries.
Image: Alexion’s new global headquarters. Photo: courtesy of Alexion.