ZMA001, in development for pulmonary arterial hypertension, has been designated as an Orphan Drug by the US Food and Drug Administration.
CI’s Three Key Takeaways:
- FDA Orphan Drug Designation: Zymedi’s ZMA001, an antibody treatment for pulmonary arterial hypertension (PAH), has been granted Orphan Drug status by the US Food and Drug Administration, providing benefits such as tax credits for clinical trial costs and seven years of market exclusivity upon approval.
- Innovative Mechanism: ZMA001 targets the infiltration of inflammation-inducing macrophages into the lungs, addressing PAH from its early stages by inhibiting symptoms fundamentally, which has shown superior efficacy in preclinical animal model studies compared to existing treatments.
- Clinical Development: Phase 1a clinical trials for ZMA001 began in January at the NIH Clinical Center, focusing on evaluating the safety and tolerability of the drug in healthy adult participants, with preliminary results to inform dosage and potential side effects for subsequent trial phases.
South Korea-based biopharmaceutical company Zymedi announced that its first-in-class antibody treatment ZMA001, currently in development for pulmonary arterial hypertension, has been designated as an Orphan Drug by the US 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.
Currently, most treatments involve vasodilators, which only alleviate symptoms without providing a cure. Due to the diverse causes of pulmonary arterial hypertension, there is a critical need for the development of targeted therapies.
ZMA001 for Pulmonary Arterial Hypertension
According to Zymedi, ZMA001 is a human monoclonal antibody that blocks the infiltration of inflammation-inducing macrophages into the lungs, thereby inhibiting the symptoms of pulmonary arterial hypertension 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.
“With the Orphan Drug designation for ZMA001, we hope to provide a valuable treatment option for [pulmonary arterial hypertension] patients in need of new therapies,” says Zymedi’s chief technology officer Nam Hoon Kwon in a release.
Phase 1a clinical trial for ZMA001 began at the National Institute of Health’s 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.
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