By PPN Staff
Within the past week, the FDA has awarded special designations to two new gene therapy candidates for hemophilia. On Oct. 25, the FDA granted orphan drug designation and investigational new drug (IND) status to an investigational factor VIII (FVIII) gene therapy for the treatment of hemophilia A currently known as SHP654 (also designated as BAX 888, Shire).
And on Oct. 26, the agency announced that it would give breakthrough therapy designation to BioMarin’s gene therapy for