European Medicines Agency Recommends Adynovi’s Approval for Hemophilia A

The European Medicines Agency has recommended marketing authorization for Shire’s Adynovi (BAX-855) as a treatment for adults and adolescents with hemophilia A.

Approval would mean the therapy could be used twice a week to prevent bleeding episodes and to counter bleeding when episodes do occur. The U.S. Food and Drug Administration has already approved the treatment under the brand name Adynovate.

The marketing recommendation from the European Medicine Agency’s Committee for Medicinal Products for Human Use means the question now goes to the European Commission for a final decision.

Adynovi is based on Advate, a drug that 69 countries have approved. They include the United States and 28 nations in the European Union.

Like Advate, Adynovi is artificially produced Factor VIII, a protein required for blood clotting that is defective in hemophilia A patients. Shire designed Adynovi to stay in the blood longer than Advate. This means doctors can reduce the frequency of injections they give patients to prevent bleeding episodes.

Both drugs are also designed to control bleeding when episodes occur.

The European Medicines Agency’s decision to recommend Adynovi’s approval was based on three Phase 3 studies (NCT01913405, NCT02210091, and NCT01736475) in hemophilia A. One trial dealt with patients having surgery. Another involved patients up to 11 years old. And the third covered patients aged 12 to 65 years.

The results of the study in adults and adolescents, titled “Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A,” was published in the journal Blood in 2015.

The European Medicines Agency decision “marks an important step in providing adults and adolescents with hemophilia A in Europe with the potential for a new treatment option with a twice-weekly prophylaxis [preventive] dosing schedule, so patients can manage their hemophilia A care with their physicians in the way that works best for them,” Dr. Howard B. Mayer, Shire’s acting director of Global Research and Development, said in a press release.

Baxalta developed Adynovate. Shire, a biotechnology company that creates treatments for rare diseases, acquired Baxalta. Shire’s portfolio includes blood diseases, immunology, genetic diseases, neuroscience, and internal medicine. It has also begun developing therapies for eye diseases and cancer.