FDA Grants Orphan-Drug Designation for Hemophilia Gene Therapy

The FDA granted orphan-drug designation to the adeno-associated virus stereotype vector SHP654 (also known as BAX 888), an investigational factor VIII (FVIII) gene therapy for the treatment of hemophilia A. The investigational therapy is intended to provide a long-acting, constant level of factor expression over several years and eliminate the need for factor-replacement therapy. A phase I/II study of SHP654 is expected to begin in the late 2017.

Source: Shire press release, October 25, 2017.

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