Mezzion Announces Re-Submission of New Drug Application for its Orphan Drug Udenafil to Treat Patients who have undergone the Fontan Operation for Single Ventricle Heart Disease
SEOUL, South Korea, March 28, 2021 /PRNewswire/ — Mezzion Pharma Co. Ltd. (140410.KQ), announced today that it has re-submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of udenafil to improve the exercise capacity of patients 12 years of age and older with single ventricle heart disease (SVHD) who have undergone the Fontan operation. Udenafil is a long acting highly selective phosphodiesterase-5 inhibitor that is orally administered. The re-submitted NDA includes a request for Priority Review, which, if granted, may shorten the FDA’s review of the re-submitted NDA to six months from the time of filing, versus a standard review timeline of 10 months from filing.
The NDA re-submission, which totals nearly 100,000 pages, is supported by data from more than 700 documents including more than 200 studies that Mezzion has completed during the last 2 decades since its founding in 2002. The re-submitted NDA package includes a pivotal Phase 3 clinical study conducted globally in close collaboration with the Pediatric Heart Network (PHN), which is funded by a grant from the National Heart, Lung, and Blood Institute (NHLBI). This was the largest pharmacotherapeutic study ever to be conducted in those with a congenital heart condition and involved the recruitment and treatment of 400 male and female adolescents from 30 PHN and auxiliary sites throughout the U.S.A., Canada, and the Republic of Korea. The results of this study, the Fontan Udenafil Exercise Longitudinal (FUEL) trial, were published in Circulation (February, 2020) abstract #20942
See also, https://www.ahajournals.org/doi/10.1161/CIRCULATIONAHA.119.044352
In 2019, the American Heart Association (AHA) recognized Mezzion’s FUEL trial as one of the most impactful discoveries and biggest victories achieved in the congenital heart disease (CHD) space that year. See https://newsroom.heart.org/news/top-heart-disease-and-stroke-research-advances-in-2019.
The results of the landmark FUEL trial demonstrated statistically significant improvements in multiple measures of exercise performance and cardiac function in patients treated with udenafil who had undergone the Fontan operation. The improvements in exercise were most pronounced at moderate levels of activity while the improvement in cardiac function was determined using the myocardial performance index, a standard echocardiographic measurement technique.
Dr. James Yeager, Chief Operating Officer of Mezzion Pharmaceuticals Inc., commented, “This NDA-resubmission was a large-scale comprehensive global effort coordinated with all FUEL study sites, study site personnel, Mezzion’s CRO and numerous consultants to verify and reformat various coding issues in the safety data base that were raised by the FDA. With those coding issues resolved and this re-submission now behind us, Mezzion expects to receive a positive review of its resubmission and FDA approval of udenafil in the last quarter of this year. If approved by the FDA, udenafil would provide the first and only approved therapy for this underserved population of patients.”
Mezzion looks forward to the FDA review process and to bringing a novel pharmacotherapeutic option to this unique Fontan patient population.
About the Fontan Procedure and Subsequent Expectations
The Fontan procedure is a surgical intervention that allows for the survival of children born with congenital heart disease characterized by only a single functional pumping chamber. This procedure consists of re-configuring the circulation to allow the single ventricle to pump blood to the body while connecting the great veins directly to the arteries that bring blood to the lungs. In this “Fontan circulation” the blood returning from the body bypasses the heart and travels to the lungs without the assistance of a dedicated right ventricle pumping chamber. The goal of the Fontan procedure is to separate the systemic and pulmonary circulations and to improve oxygen levels by redirecting venous blood directly to the lungs.
While the Fontan procedure creates a stable circulation, the risk of hospitalization and cardiac death rises significantly in the second and third decades after Fontan completion, a risk that is associated with a decline in exercise capacity. The Fontan circulation is also associated with non-cardiac complications such as protein-losing enteropathy, plastic bronchitis, and liver failure, all of which can be attributed to a chronic elevation in central venous pressure and a chronically reduced cardiac output. For all of these reasons, a 35 year-old patient who has gone through Fontan palliation has the approximate life expectancy of a 75 year old with normal cardiac physiology.
Mezzion Pharma Co., Ltd.
Mezzion Pharma Co., Ltd. is headquartered in Korea. Mezzion and its wholly owned subsidiary, Mezzion Pharmaceuticals, Inc., have administrative offices in Deerfield, Illinois and Boca Raton, Florida. Mezzion Pharma is an innovation-driven pharmaceutical company that is focused on discovering, developing, and commercializing novel therapeutics in the field of rare pediatric diseases. Mezzion Pharma is a publicly-listed pharmaceutical company in Korea on the Korean stock exchange under (140410:KOSDAQ).
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding: Mezzion Pharma’s expectations regarding the potential benefits of udenafil; Mezzion Pharma’s expectations regarding the anticipated timing of any future clinical trials; Mezzion Pharma’s expectations on regulatory submissions for marketing approval of udenafil for the treatment of patients that have undergone the Fontan operation, to improve exercise capacity in the United States, including the timing of these submissions; and Mezzion Pharma’s expectations regarding the potential commercial launch of udenafil, including the timing of a potential approval of udenafil. Risks and uncertainties that contribute to the uncertain nature of the forward-looking statements include: the expectation that Mezzion Pharma will need additional funds to finance its operations; Mezzion Pharma’s or any of its collaborative partners’ ability to initiate and/or complete clinical trials; the unpredictability of the regulatory process; the possibility that Mezzion Pharma’s or any of its clinical trials will not be successful; Mezzion Pharma’s dependence on the success of udenafil; Mezzion Pharma’s reliance on third parties for the manufacture of Mezzion Pharma’s udenafil and udenafil tablets; possible regulatory developments in the United States and foreign countries; and Mezzion Pharma’s ability to attract and retain senior management personnel.
These and other risks and uncertainties are described more fully in Mezzion Pharma’s most recent filings with the 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 Mezzion Pharma. All forward-looking statements contained in this press release speak only as of the date on which they were made. Mezzion undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.
SOURCE Mezzion Pharma Co. Ltd.
Posted: March 2021
- Mezzion Announces Submission of New Drug Application for its Orphan Drug Udenafil to Treat Patients who have undergone the Fontan Operation for Single Ventricle Heart Disease – June 30, 2020
udenafil FDA Approval History
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