SEOUL, South Korea and BETHESDA, Md., June 22, 2018 /PRNewswire/ -- Mezzion Pharma and the National Heart, Lung, and Blood Institute (NHLBI) today announced that it has completed enrollment of their FUEL trial, a pivotal Phase 3 efficacy and safety trial of udenafil for the treatment of adolescents with congenital single ventricle heart disease.
On June 14th, this landmark study reached its enrollment target of 400 participants. This randomized, double-blind, placebo-controlled clinical trial is evaluating the safety and efficacy of orally administered udenafil in adolescents with a single functional ventricle who had previously undergone Fontan surgical palliation. The study enrolled 400 male and female adolescent participants in total at 24 Pediatric Heart Network http://www.pediatricheartnetwork.org/ (PHN) and auxiliary sites throughout the U.S., Canada, and Korea. All participants in the FUEL trial as well as naïve Fontan participants are eligible to participate in an open-label extension study (OLE), which is currently recruiting and enrolling.
Mezzion Pharma and the PHN investigators would like to acknowledge the enormous and selfless commitment on the part of the participants and their families. Concluding enrollment of this landmark trial represents a significant achievement in our efforts to develop a disease-modifying treatment that we hope will advance the standard of care and improve the quality of life for adolescents born with congenital single ventricle heart disease who have undergone Fontan palliation.
"Mezzion is extremely thankful and grateful for the courageous Fontan subjects and their families that participated in the FUEL trial. Meeting the enrollment goal was an extraordinary effort by the PHN site personnel and the NHLBI," stated James Yeager, RPh., Ph.D., Chief Scientific Officer and Director of Mezzion Pharma Co., Ltd. "We look forward with excitement to completion of the FUEL trial and announcing the top-line data and NDA filing with the FDA in 2019. If approved, it will make udenafil the first drug ever approved by the FDA for treating adolescents born with congenital single ventricle heart disease who have undergone Fontan palliation."
The clinical program for this orphan drug indication is using protocols developed by PHN which is funded by NHLBI, an Institute of the National Institutes of Health (NIH). The Phase 3 protocols are being conducted under special protocol assessment (SPA) review by the FDA and were found sufficient to support a regulatory submission for the orphan drug indication, provided that all statutory requirements for drug approval are met. Having an FDA-approved drug with such an indication would be a first for patients with congenital single ventricle heart disease with Fontan physiology.
The primary efficacy outcome being studied is improvement in exercise capacity as measured by the change in maximal VO2 after 6 months of daily therapy compared to placebo. People who have had the Fontan procedure often find that their ability to exercise decreases significantly during their teenage years, so preventing this decline would be an important clinical outcome. There are also a number of the secondary endpoints, including safety assessments.
The Fontan procedure is the final surgery in a series of palliative surgical operations in children born with single ventricle congenital heart disease. Fontan patients have a significantly shortened life span, often not surviving past the third or fourth decade of life, due to factors, such as, deteriorating ventricular performance and associated secondary pathologies. Mezzion Pharma estimates that there are approximately 29,000 persons alive today in the U.S. with this disease.
Mezzion Pharma Co. Ltd.
Mezzion Pharma is a publicly-listed biotech company in Korea focusing on the development of new drugs.
Statements under the Private Securities Litigation Reform Act: with the exception of the historical information contained in this release, the matters described herein contain forward-looking statements that involve risk and uncertainties that may individually or mutually impact the matters herein described, including but not limited to FDA review and approval, product development and acceptance, manufacturing, competition, and/or other factors, which are outside the control of the Company.
Mr. Won Geun KIM
Tel: +82 2 560 8008
Email: [email protected]
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