Gamida Cell, a leading cellular and immune therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for NiCord as a treatment for hematopoietic stem cell transplantation (HSCT).
Gamida Cell was previously granted orphan drug designation for NiCord by the FDA as a treatment for several hematologic malignancies, and by the European Medicines Agency (EMA) as a treatment for HSCT.
The FDA grants orphan drug designation to investigational drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug status is intended to facilitate drug development and may provide benefits to drug developers, including up to seven years of market exclusivity upon regulatory product approval and exemptions from certain FDA application fees.
