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Agomab Secures FDA Orphan Drug Status for AGMB-447 to Address Idiopathic Pulmonary Fibrosis

Friday, June 07, 2024

Agomab Therapeutics NV announced today that its inhaled small molecule inhibitor of ALK5, AGMB-447, has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA). The company is currently evaluating AGMB-447 in a Phase 1 clinical trial (NCT06181370) as a potential treatment for Idiopathic Pulmonary Fibrosis (IPF).

The FDA's Orphan Drug Designation program supports the development of treatments for rare diseases affecting fewer than 200,000 people in the U.S. This designation provides various benefits, including market exclusivity and financial incentives such as tax relief for clinical research costs.

Agomab Therapeutics, stated, “The FDA's Orphan Drug Designation for AGMB-447 reinforces our belief in its potential to provide meaningful therapeutic benefits for IPF patients.” He expressed optimism about advancing through the ongoing Phase 1 trial, particularly in evaluating data from single and multiple ascending dose studies in healthy subjects and IPF patients.

It should be noted that AGMB-447 is still investigational and has not been approved by any regulatory authority. Therefore, its safety and effectiveness have not been confirmed.

AGMB-447 is a lung-restricted small molecule inhibitor targeting ALK5 (also known as TGFβRI), developed for the treatment of IPF and other fibrotic respiratory conditions. IPF is a debilitating disease affecting approximately 100,000 individuals in the U.S., characterized by the excessive accumulation of fibrotic tissue in the lungs, resulting in lung stiffness, impaired breathing, and reduced oxygen absorption. Despite available treatments, the average survival rate post-diagnosis is only three to five years without a lung transplant. TGFβ is recognized as a key regulator of fibrosis in IPF, and initial clinical data suggests that targeting this pathway could be beneficial. AGMB-447 is designed to effectively inhibit ALK5 in the lungs while being rapidly metabolized in the bloodstream to minimize systemic exposure.



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