Drugs Xagena
The European Medicines Agency ( EMA ) has recommended granting a marketing authorisation under exceptional circumstances for Strensiq ( Asfotase alfa ), for the long-term treatment of hypophosphatasia, a rare inherited metabolic disorder affecting the bones, in patients who have developed the disease in childhood.
Hypophosphatasia is caused by defects in the gene responsible for producing alkaline phosphatise ( ALP ), an enzyme that plays a key role in creating and maintaining healthy bones, and managing calcium and phosphate in the body.
Patients with hypophosphatasia have symptoms such as early loss of teeth, malformed bones and frequent bone fractures.
This disease can be life-threatening when it affects unborn babies or infants, because of the incomplete development of their bones and additional complications such as respiratory problems. When it develops later in life, the disease is generally not
fatal but can be highly debilitating.
There is currently no approved treatment for this condition; patients usually receive supportive treatment such as plaster casts for broken bones, calcium supplements for maintaining the levels of calcium in the blood and painkillers.
Strensiq, the first therapy for this disease, could contribute to respond to this unmet medical need as it is expected to help improve the composition of bones and make them stronger.
Asfotase alfa, its active substance, is a modified copy of the human ALP enzyme and serves as a replacement for the defective enzyme.
Because hypophosphatasia is rare, Strensiq was also designated as an orphan medicine by the Committee for Orphan Medicinal Products ( COMP ) in 2008. ( Xagena )
Source: EMA; 2015
XagenaMedicine_2015
