The first treatment in Europe available for patients with the rare bone disease hypophosphatasia is a step closer following a recommendation from the medicines safety watchdog.
The European Medicines Agency (EMA) is proposing that the orphan medicine asfotase alfa (Alexion Europe SAS’s Strensiq) should be granted an exceptional circumstances marketing authorisation for hypophosphatasia.
The condition is caused by a genetic disorder which affects the development of healthy bones and the body’s management of calcium and phosphate. It can be life threatening in unborn babies or infants and debilitating for others when it develops in later life, according to the EMA.
Asfotase alfa is a modified copy of the human alkaline phosphatase enzyme and acts as a replacement for the defective enzyme. When administered, it is expected to improve the composition of the bones of people with this condition and make them stronger.
The product will be made available in the first instance to young children and adolescents and will also be offered to them as they become older, the EMA says.
It will be available in different strengths and volumes and will be colour coded to distinguish between the different doses. Advice booklets for patients and their carers will be produced, according to the EMA.
The recommendation to approve asfostase alfa now goes to the European Commission for approval. It will then be left to member states to decide on its price and reimbursement within their own healthcare system.