FDA Grants Orphan Drug Designation (ODD) Status to Zymedi's ZMA001 for Pulmonary Arterial Hypertension

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Jul 30, 2024, 20:00 ET

INCHEON, South Korea, July 30, 2024 /PRNewswire/ -- Zymedi (CEO Sunghoon Kim) announced that its first-in-class antibody treatment ZMA001, currently in development for pulmonary arterial hypertension (PAH), has been designated as an Orphan Drug by the U.S. Food and Drug Administration (FDA).

Pulmonary arterial hypertension is an inflammatory vascular disease characterized by the remodeling and narrowing of pulmonary arterioles due to various causes. The infiltration of immune cells from the blood into the lungs damages the pulmonary system, increases pulmonary arterial pressure, and ultimately leads to right ventricular failure and death, with a survival rate comparable to cancer.

Initially manifesting as shortness of breath, PAH progresses to chest pain, fainting, and extreme fatigue, severely impacting daily life. Globally, approximately 70,000 people are affected by PAH, with around 6,000 cases in South Korea, making it a rare disease.

Currently, most treatments involve vasodilators, which only alleviate symptoms without providing a cure. Due to the diverse causes of PAH, there is a critical need for the development of targeted therapies.

According to Zymedi, ZMA001 is a human monoclonal antibody that blocks the infiltration of inflammation-inducing macrophages into the lungs, thereby fundamentally inhibiting the symptoms of PAH from the early stages. In preclinical development, animal model studies demonstrated superior efficacy compared to existing drugs.

Notably, ZMA001 has shown a synergistic effect when used in conjunction with existing medications, positioning it as a potential game-changer in PAH treatment, where innovative drugs are urgently needed.

Zymedi's CTO Nam Hoon Kwon stated, "With the Orphan Drug designation for ZMA001, we hope to provide a valuable treatment option for PAH patients in need of new therapies."

Kwon further emphasized, "The FDA's Orphan Drug designation offers various benefits, including tax credits for clinical trial costs, seven years of market exclusivity upon approval, and assistance with the drug development process, which are particularly advantageous for startup companies with limited resources and time constraints, aiding in clinical progression and commercial success."

Phase 1a clinical trial for ZMA001 began at the NIH Clinical Center in January, involving healthy adult participants. This initial phase focuses on evaluating the safety and tolerability of the drug in humans. Preliminary results from these trials will help determine the appropriate dosages and identify any potential side effects, paving the way for subsequent phases of clinical testing.

About Zymedi

Zymedi is a global biopharmaceutical company that is committed to transforming the role of ARSs (Aminoacyl-tRNA Synthetases) and targeting it as a method of discovering and developing innovative treatments for patients with serious and life-threatening conditions that have no standard of care or lack effective therapy.

For more information, please visit https://zymedi.com/

SOURCE Zymedi