EMA Recommends First Hypophosphatasia Medicine

Miriam E Tucker


June 26, 2015

The European Medicines Agency (EMA) has recommended marketing authorization of asfotase alfa (Strensiq, Alexion Pharma) as the first-ever treatment for patients who develop hypophosphatasia in childhood.

Hypophosphatasia is an inherited bone disorder caused by mutations in the alkaline phosphatase gene. Symptoms include early loss of teeth, malformed bones, and frequent bone fractures. This disease can be life-threatening when it affects unborn babies or infants.

Current treatments are supportive, including plaster casts for broken bones, calcium supplements, and painkillers.

Asfotase alfa, a modified copy of the human alkaline phosphatase enzyme that serves as a replacement for the defective enzyme, is expected to improve strength and composition of bones. The medicine is to be used in patients who develop symptoms in childhood and may also be used later in their adult life.

It will be available in various strengths and volumes so that doses can be adjusted to the patient's size and weight. The EMA has recommended a color coding system to allow for distinguishing among the different forms. Information booklets will be available for patients and caregivers.

Because hypophosphatasia is so rare, few data are available on the efficacy and safety of asfotase alfa. For that reason, the EMA's Committee for Medicinal Products for Human Use (CHMP) said that the medicine should be recommended for marketing authorization under exceptional circumstances, a type of authorization granted for medicines that offer new or improved treatment options for patients with no or only limited alternatives, in cases where the manufacturer can't provide comprehensive data.

However, CHMP will require Alexion to collect further data on its clinical efficacy and safety and submit these data regularly for review following marketing authorization.

Asfotase alfa was also designated as an orphan medicine by the Committee for Orphan Medicinal Products (COMP) in 2008. Orphan designation provides medicine developers access to incentives such as fee reductions for scientific advice, with the aim of encouraging the development of treatments for rare disorders.

The CHMP opinion will now be sent to the European Commission for the adoption of a decision on a European Union–wide marketing authorization. Once that has been granted, each member state will make a decision on price and reimbursement based on the potential role/use of this medicine in the context of its national health system.

The US Food and Drug Administration is expected to make its approval decision for this drug by July 29.

European Medicines Agency. June 26, 2015. Press release


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