Inhibrx, Inc. announced that the U.S. Food and Drug Administration, or FDA, has granted orphan-drug designation for INBRX-101 for the treatment of alpha-1 antitrypsin deficiency, or AATD.
“We believe this designation from the FDA acknowledges the significant unmet need for novel therapeutic options in a patient community where the standard of care has not seen an improvement in many decades,” said Inhibrx Chief Executive Officer, Mark Lappe. “We believe INBRX-101 can greatly improve the quality of life for AAT patients and look forward to working closely with regulators, key opinion leaders, patients and their families to achieve this.”
The FDA’s Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases, or conditions that affect fewer than 200,000 people in the U.S. Orphan-drug designation provides certain benefits, including financial incentives to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval.