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



Treatment for Single Ventricle Heart Disease

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

SEOUL, South Korea, June 30, 2020 /PRNewswire/ – Mezzion Pharma Co. Ltd. (140410.KQ), announced today that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of udenafil to improve the physiology 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 NDA includes a request for Priority Review, which, if granted, may shorten the FDA’s review of the NDA to six months from the time of filing, versus a standard review timeline of 10 months from filing.

The NDA 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 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 (December 2019) abstract #20942 .

The American Heart Association (AHA) recognized Mezzion’s FUEL trial as the biggest victory in congenital heart disease (CHD) advancement for 2019. Dr. Stephen Paridon, the principal investigator of the FUEL trial had the following comment, “The unique collaboration between Mezzion and the PHN allowed for the successful completion of the FUEL trial, a study that demonstrated an important effect of udenafil on exercise performance and heart function in those born with SVHD.”

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 large-scale Phase 3 study for an orphan disease indication using a drug designated for a rare pediatric disease took five years to complete and was challenging to execute. We owe a major debt of gratitude to all of the patients who participated in the study, their parents and the clinical investigators who led the study. Our thanks also go out to the PHN and the NHLBI along with a countless number of consultants, contract research and manufacturing organization partners. 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.”   

Jodi Smith, Esq., Program Director for Mended Hearts and Mended Little Hearts, a USA-based advocacy group for patients with heart disease and their families, states, “As a parent of a child with Fontan palliation who had participated in this study, I am encouraged to see the results that show improvement in day-to-day life activities. I have seen this improvement firsthand in my son. I am also glad to see that the clinical data seem to show improvement in numbers that impact heart function and decrease heart failure. All of this, together, can improve the quality of life for single-ventricle patients like my son.”

Mezzion looks forward to the FDA review process and to bringing a novel pharmacotherapeutic option to this unique patient population. Dr. David Goldberg, co-Principal Investigator summed it up nicely. “After years of borrowing medical therapies from the adult heart failure experience, we finally have a drug that specifically targets the unique physiology of SVHD after Fontan palliation.”

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 presence of a 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).

Forward-Looking Statements

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: June 2020

udenafil FDA Approval History

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