Mezzion Pharma Receives Clear FDA Path Forward for the Approval of Udenafil for Single Ventricle Heart Disease
SEOUL, South Korea and BETHESDA, Md. , May 31, 2022 /PRNewswire/ -- Mezzion Pharma Co., Ltd. (Mezzion Pharma) met with the FDA Division of Cardiology and Nephrology (DCN) on Friday, May 27th to discuss with the FDA the path forward for the regulatory approval of udenafil for the treatment of single ventricle heart disease (SVHD) in children with Fontan physiology. More specifically, Mezzion sought clarity on the critical elements of an additional clinical trial that may be needed to complete the NDA submission.
The FDA responded to the Meeting Background Package that was submitted by Mezzion to DCN with detailed advice and direction for the path forward for an FDA approval. Specifically, only one new clinical trial, FUEL-2, will be needed. FUEL-2 will be similar to the previously completed FUEL-1 trial (Double-Blind, Placebo Controlled Safety and Efficacy Trial) in the adolescent and young adult population. The primary difference in FUEL-2 will be the exclusion of 'Super Fontan' patients with peak VO₂ ≥ 80% of predicted to avoid de-enriching the FUEL-2 study population. We learned from FUEL-1 that 'Super Fontan" patients are at near-maximum physiologic value and, thus, their peak VO2 cannot be meaningfully further improved. We therefore learned from FUEL-1 that, when the 'Super Fontan' patients were excluded (n=300) from the FUEL-1 study population, the subgroup met both the primary (p-value=0.023) and secondary endpoints. We are therefore confident that we will achieve similar statistical significance for the primary and secondary endpoints in FUEL-2 with an enriched target population.
If the additional new trial meets statistical significance on the primary endpoint, then FDA agreed that it will be considered a positive trial and sufficient for approval. The FDA also advised that meeting statistical significance on the secondary endpoints, while enhancing the potential labeling for the product, would not be a prerequisite for approval. Mezzion anticipates enrolling the first patient within the next few months.
The FDA agreed with Mezzion regarding the specific primary and secondary endpoints to use for the measurement of function capacity in trial subjects in the new trial, FUEL-2, and further agreed that the protocol for this new trial would be reviewed by DCN under a Special Protocol Assessment.
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, but serious age-advanced, 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.
Contact:
Dr. James Yeager, Deerfield, Illinois, USA, Tel: +1-847-2122679
Email: [email protected]
Mr. S.I. Noh, Seoul, Korea, Tel: +82 2 560 8000
Email: [email protected]
SOURCE Mezzion Pharma Co. Ltd.
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