Saturday, June 15, 2024

Pulmonary Fibrosis Treatment: FDA Grants Orphan Drug Designation to Agomab’s AGMB-447

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Pulmonary fibrosis treatment by Agomab Therapeutics NV (Agomab) has received an Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for its investigational drug AGMB-447. This inhaled, small molecule inhibitor of ALK5 is currently being evaluated as a potential treatment for idiopathic pulmonary fibrosis (IPF) in a Phase 1 clinical trial (NCT06181370).

The FDA’s Orphan Drug Designation program is intended to facilitate the development of medicinal treatments for rare diseases affecting fewer than 200,000 people in the United States. This designation provides several benefits, including market exclusivity, tax credits for clinical research, and a waiver of certain FDA fees, which can be pivotal for companies developing treatments for rare conditions.

“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 for 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 idiopathic pulmonary fibrosis patients.”

Promising Investigational Drug for Idiopathic Pulmonary Fibrosis

AGMB-447 is an investigational drug and is not yet approved by any regulatory authority. Its efficacy and safety have yet to be established through clinical trials. AGMB-447 is a small molecule lung-restricted inhibitor of ALK5, also known as TGFβRI. It is designed to treat idiopathic pulmonary fibrosis and other fibrotic respiratory conditions. IPF is a serious disease affecting approximately 100,000 patients in the U.S.

It is characterized by the uncontrolled production of fibrotic, scar-like tissue that accumulates in the lungs. This buildup makes the lungs stiff, impeding breathing and reducing the efficiency of oxygen absorption into the blood. Although some medicinal treatments exist, the average survival time after diagnosis is only three to five years without a lung transplant.

Transforming Growth Factor β (TGFβ) is recognized as a key regulator of fibrosis in idiopathic pulmonary fibrosis, and preliminary clinical data supports the targeting of this pathway. AGMB-447 is specifically engineered to inhibit ALK5 effectively and safely within the lungs. This is achieved through its rapid metabolism via hydrolysis in the plasma, preventing significant systemic exposure and thereby reducing the potential for side effects.

Pulmonary Fibrosis

Agomab Therapeutics Targets Fibrosis and Regeneration in Chronic Conditions

Agomab Therapeutics is dedicated to achieving disease modification by targeting fibrosis and regeneration in chronic conditions such as fibrostenosing Crohn’s disease and idiopathic pulmonary fibrosis. The company focuses on biologically validated pathways, including Transforming Growth Factor β and Hepatocyte Growth Factor, utilizing specialized capabilities in organ-restricted small molecules and high-affinity antibodies.

With a differentiated clinical pipeline addressing several fibrotic disorders, Agomab combines end-to-end research and development capabilities with a proven business development track record. Supported by a strong investor base, Agomab is establishing itself as a leading European biopharmaceutical company.

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The FDA’s Orphan Drug Designation is a crucial milestone for Agomab, offering significant incentives that can accelerate the development of AGMB-447. Market exclusivity ensures that, upon approval, Agomab would have a seven-year period during which no direct competitor could market a similar drug for the same indication. Tax credits for clinical research costs provide financial relief, making it more feasible for Agomab to continue their essential studies. The waiver of FDA fees further reduces the financial burden on the company, allowing more resources to be directed toward research and development.

FDA Grants Orphan Drug Status to AGMB-447 for Idiopathic Pulmonary Fibrosis

The designation also underscores the potential of AGMB-447 to provide a new therapeutic option for patients with idiopathic pulmonary fibrosis, a disease with limited treatment options and a high mortality rate. The current phase of the clinical trial is crucial in determining the safety and efficacy of AGMB-447. Positive results could lead to further clinical development and eventually offer a new hope for patients suffering from this debilitating disease.

As Agomab continues its Phase 1 clinical trial, the medical community eagerly anticipates the data from the single ascending dose and multiple ascending dose evaluations. These results will provide critical insights into the drug’s safety profile and its potential effectiveness in treating idiopathic pulmonary fibrosis. Agomab’s commitment to targeting biologically validated pathways and its focus on organ-specific treatments reflect a strategic approach to addressing complex chronic conditions. The successful development of AGMB-447 could pave the way for new treatments in the realm of fibrotic diseases, significantly impacting patient care and outcomes.

In conclusion, the FDA’s Orphan Drug Designation for AGMB-447 marks a significant step forward in the treatment of Idiopathic Pulmonary Fibrosis. Agomab’s innovative approach and robust clinical development program position it well to potentially offer new hope to patients with this challenging condition.


Resource: Agomab Therapeutics, June 06, 2024

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