Potential Gene Therapy GENV-HEM Earns Orphan Drug Status

Potential Gene Therapy GENV-HEM Earns Orphan Drug Status

311081

Potential Gene Therapy GENV-HEM Earns Orphan Drug Status

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to GENV-HEM, GeneVentiv Therapeutics’ investigational gene therapy for hemophilia A and B patients, with or without clotting factor inhibitors. Orphan drug designation is given to treatment candidates that have the potential to be safe and effective treating rare diseases, defined as those affecting fewer than 200,000 people in the U.S. The decision is meant to expedite GENV-HEM’s clinical development by providing regulatory support…

You must be logged in to read/download the full post.