Mezzion Pharma said its experimental drug, udenafil, improved exercise capacity in adolescents with single ventricular congenital heart disease (SVHD) in a global phase 3 study.
The most recent data did not reach clinical significance in the traditional measurement, which evaluates the maximal oxygen uptake with the subject's maximal effort (VO2 peak). Still, it successfully achieved oxygen consumption at the ventilatory anaerobic threshold (VO2 with VAT), the company said.
With the findings, Mezzion said he would send a new drug order (NDA) to the US Food and Drug Administration.
The Kosdaq-listed company presented key findings from the phase 3 study of udenafil for the treatment of SVHD at the American Heart Association (AHA) Annual Scientific Session in Philadelphia, USA, on Sunday.
Dr. David Goldberg, a pediatric cardiologist at the Children's Hospital of Philadelphia, presented the data at a symposium entitled "Fontan Physiology and Results of the FUEL Study."
Udenafil aims to improve heart function and exercise capacity in adolescents who underwent Fontan surgery due to SVHD. The phase 3 FUEL study evaluated the efficacy and safety of udenafil in 400 patients for six months.
The main objective of the FUEL study was to improve exercise capacity at 26 weeks after treatment by measuring changes in VO2 peak or maximal oxygen uptake at the subject's maximal effort. Results showed that peak VO2 of the udenafil group improved 3.2% at week 26 compared with baseline. The result showed improvement compared to 0% of the placebo group, but did not reach clinical significance.
However, the results indicated that VO2 in IVA, which was more appropriate than peak VO2 to measure exercise capacity, showed significant improvement in the study, according to Mezzion.
Dr. Stephen Paridon, pediatric cardiologist and exercise physiologist at Children's Hospital of Philadelphia, explained that Fontan patients do not have a ventricle to pump venous blood to the lung and that their blood flow to the lung depends only on central venous pressure in the body.
As central venous pressure is already elevated at baseline, they find it difficult to achieve full exercise capacity, he added, adding that peak VO2 may not be the best measure of Fontan patients' exercise capacity.
Instead, VO2 in VAT could be a more relevant measure because central venous pressure limits it less, he noted.
VO2 in IVA improved by 2.9% in the udenafil group compared with a 1.0% decline in the placebo group. It was also statistically significant, Mezzion said.
The company had a meeting with the FDA's cardiovascular and renal division to discuss key study data in October.
"The FDA has confirmed that Mezzion can proceed with the NDA submission to udenafil to improve the exercise capacity of individuals with SVHD and that the submission would be" archivable "as long as the submission contained all necessary information." said Mezzion.
Based on the positive results from the FUEL study and udenafil safety data, the company would quickly send an NDA to the FDA to get approval for udenafil use, he added.
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