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AliveGen's ALG-801 Receives FDA Orphan Drug Designation for Pulmonary Arterial Hypertension

• AliveGen's ALG-801 has been granted Orphan Drug Designation by the FDA for treating pulmonary arterial hypertension (PAH). • The designation highlights the urgent need for improved treatments for this rare and life-threatening condition. • ALG-801, a novel ActRIIA/IIB hybrid ligand trap, has shown promising efficacy and safety in Phase 1 studies. • This designation provides AliveGen with benefits including tax credits and potential marketing exclusivity.

AliveGen USA Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ALG-801 for the treatment of pulmonary arterial hypertension (PAH). PAH is a rare, serious, and life-threatening condition characterized by high blood pressure in the lung arteries, potentially leading to right-sided heart failure.

Addressing Unmet Needs in PAH Treatment

"FDA’s Orphan Drug Designation for ALG-801 underscores the need for identifying better treatment options for patients suffering from PAH," said Dr. HQ Han, CEO of AliveGen. He added that this designation marks a significant step in AliveGen’s mission to develop first-in-class/best-in-class therapeutics for conditions with high unmet medical needs. The company believes ALG-801 has the potential to become a best-in-class medicine for PAH, citing its superior efficacy and improved safety profile demonstrated in preclinical and Phase 1 clinical studies.

Benefits of Orphan Drug Designation

The FDA's Orphan Drug Designation program supports the development of drugs and biologics for rare diseases affecting fewer than 200,000 people in the United States. The designation offers several benefits to sponsors, including tax credits for clinical trial costs, exemptions from certain FDA fees, and the potential for seven years of market exclusivity upon approval.

About ALG-801

ALG-801 is a novel, next-generation ActRIIA/IIB hybrid ligand trap. It is designed to selectively sequester a specific set of Smad2/3 pathway-activating ligands, which play a critical role in the pathogenesis of PAH and other diseases. ALG-801 has successfully completed Phase 1a and 1b clinical trials in healthy volunteers and is now ready for Phase 2 clinical development.

AliveGen's Focus

AliveGen is a clinical-stage biotechnology company based in Thousand Oaks, California, focused on discovering first-in-class/best-in-class biotherapeutics for diseases with high unmet medical needs, including cardiometabolic disorders, neuromuscular diseases, age-related osteosarcopenia, and various wasting disorders. The company aims to improve patient care and quality of life through innovative and effective therapies.
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Reference News

[1]
AliveGen Receives FDA Orphan Drug Designation for ALG-801 for Pulmonary Arterial Hypertension
morningstar.com · Jan 7, 2025

AliveGen USA Inc. received FDA Orphan Drug Designation for ALG-801, targeting pulmonary arterial hypertension (PAH). ALG...

[2]
AliveGen Receives FDA Orphan Drug Designation for ALG-801 for Pulmonary Arterial Hypertension
finance.yahoo.com · Jan 7, 2025

AliveGen USA Inc. received FDA Orphan Drug Designation for ALG-801, targeting pulmonary arterial hypertension (PAH). ALG...

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