A New Drug Application (NDA) has been submitted to the Food and Drug Administration (FDA) for udenafil to improve the physiology of patients aged ≥12 years with single ventricle heart disease (SVHD) who have undergone the Fontan operation.
Udenafil is an orally administered, potent and selective phosphodiesterase type 5 inhibitor (PDE5). Inhibition of PDE5 results in the relaxation of pulmonary vascular smooth muscle cells and vasodilation of the pulmonary vascular bed.
The NDA is supported by data from hundreds of studies completed over the last 2 decades, including the pivotal phase 3 FUEL trial, which evaluated the efficacy and safety of udenafil in 400 adolescent patients who underwent the Fontan operation. Patients were randomized 1:1 to receive either udenafil 87.5mg orally twice daily or placebo plus standard therapy. The primary end point was the change in exercise capacity from baseline to week 26, as measured by maximal VO2 at maximum exercise effort.
Results from the trial showed that udenafil was not associated with statistically significant improvements in oxygen consumption at peak exercise, however analysis at ventilatory anaerobic threshold showed significant improvements in submaximal exercise performance. Additionally, findings of the study showed the treatment was not associated with changes in myocardial performance index, log-transformed reactive hyperemia index, or serum brain-type natriuretic peptide levels.
“This large-scale phase 3 study for an orphan disease indication using a drug designated for a rare pediatric disease took 5 years to complete and was challenging to execute,” said Dr James Yeager, Chief Operating Officer of Mezzion Pharmaceuticals. “We now look forward to the potential approval and launch of udenafil over the next year. If approved, udenafil would provide the first and only approved therapy for this underserved population of patients.”
The Company has requested Priority Review, which may shorten the FDA’s review of the application to 6 months if granted.
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