Agomab receives FDA Orphan Drug Designation for AGMB-447 in idiopathic pulmonary fibrosis

AGMB-447 is an inhaled lung-restricted ALK5-inhibitor currently in a Phase 1 clinical trial 

Agomab Therapeutics announced that it has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for AGMB-447, its inhaled, small molecule inhibitor of ALK5. Agomab is evaluating AGMB-447 as a potential treatment for Idiopathic Pulmonary Fibrosis (IPF) in a Phase 1 clinical trial.

The FDA’s Orphan Drug Designation program is designed to facilitate development of medicinal treatments for rare diseases that affect fewer than 200,000 people in the U.S. The designation provides companies with various development and commercial benefits, including market exclusivity and a range of financial incentives, such as tax relief for clinical research costs.

“Receiving Orphan Drug Designation from the FDA provides further support that AGMB-447’s mechanism of action has the potential to achieve meaningful therapeutic benefits to IPF patients,” said Philippe Wiesel, Chief Medical Officer at Agomab Therapeutics. “As we progress through our ongoing first-in-human Phase 1 trial, we look forward to evaluating the data from the single ascending dose and multiple ascending dose evaluation of AGMB-447 in healthy subjects and IPF patients,” he added.

AGMB-447 is an investigational drug and not approved by any regulatory authority. Its efficacy and safety have not been established.