FDA Designates Hemophilia Gene Therapy as Orphan Drug

Shire received an orphan-drug classification from the FDA for its gene therapy for hemophilia.

To control bleeding from hemophilia A, the therapy delivers a working copy of the gene whose defect causes the disease, which is due to an absence of a blood-clotting factor.

The goal of the therapy is to equip the body to produce its own clotting factor, instead of relying on the current therapy of continual factor replacement by external means, Shire said.