FDA Grants Orphan Drug Status to AliveGen's ALG-801 for Pulmonary Arterial Hypertension

AliveGen USA Inc., a biotechnology firm based in Thousand Oaks, California, has announced a significant development in the realm of treatments for pulmonary arterial hypertension (PAH). The U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation to ALG-801, a promising therapy for PAH—a rare and severe condition marked by elevated blood pressure in the arteries of the lungs, potentially leading to heart failure on the right side of the heart.

Dr. HQ Han, the CEO of AliveGen, emphasized the importance of this designation, noting that it highlights the critical need for improved treatment options for PAH patients. He stated that this recognition is a key milestone in AliveGen's ongoing efforts to create groundbreaking therapeutics aimed at addressing significant unmet medical needs. Dr. Han expressed confidence in ALG-801, citing its superior efficacy and enhanced safety profile demonstrated in both preclinical and Phase 1 clinical studies. He believes that the drug holds immense potential to become a leading treatment for PAH and expressed anticipation for its continued clinical development.

The FDA's Orphan Drug Designation is part of a program aimed at promoting the development of drugs and biologics intended to treat, diagnose, or prevent rare diseases affecting fewer than 200,000 individuals in the United States. This designation offers certain benefits to drug sponsors that support the development of treatments for small patient populations with unmet needs. These benefits include tax credits for clinical trial costs, waivers from some FDA fees, and the possibility of seven years of market exclusivity upon approval.

ALG-801 is described as an innovative next-generation ActRIIA/IIB hybrid ligand trap. It is engineered to selectively bind a specific group of Smad2/3 pathway-activating ligands, which play a crucial role in the development of PAH and various other diseases. Having successfully completed Phase 1a and 1b clinical trials in healthy individuals, ALG-801 is now poised to advance to Phase 2 clinical trials.

AliveGen is a privately-owned clinical-stage biotechnology company committed to discovering and developing pioneering biotherapeutics. The company's focus is on treating diseases with significant unmet medical needs, including cardiometabolic disorders, neuromuscular diseases, age-related conditions like osteosarcopenia, and various wasting disorders. AliveGen's mission is to enhance patient care and quality of life through the development of innovative, safe, and highly effective therapies.