April 26, 2024

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[THE INVESTOR] South Korea’s biotech firm Mezzion Pharma said on Jan. 13 that its late-stage clinical trials for its experimental treatment for patients with with fontan surgical palliation is running smoothly.

The company expects that some 400 patients will take part in the phase 3 clinical program by end-2017 which is designed to evaluate the use of extended half-life phosphodiesterase type 5 inhibitor udenafil to treat adolescents who have undergone the Fontan surgical palliation for single ventricle heart defects.

 

Mezzion Pharma CEO Park Dong-hyun

A total of 30 hospitals -- 27 in the US, two in South Korea and one in Canada -- have agreed to participate in the trials.

Some 100 patients at 17 US sites have been enrolled for the trials to date, the company said.

“The most important factor in the development of a rare disease treatment is how well the patient recruitment process should proceed,” an official at Mezzion Pharma said.

The studies will assess the improvement in certain acute and chronic symptoms in adolescents who had the Fontan procedure earlier in life.

On Jan. 11, the Korea Food and Drug Administration designated udenafil as a developmental-stage orphan drug for use in patients with a single functional ventricle who have undergone Fontan surgical palliation. The US Food and Drug Administration also granted orphan status to the treatment in September last year.

The Fontan operation is the final surgery in the strategy of staged palliation for children born with congenital heart defects resulting in functional single-ventricle physiology.

Fontan patients have a significantly shortened life span, often not surviving past their 30s or 40s, mainly due to deteriorating ventricular performance and increasing pulmonary vascular resistance.

By Park Han-na (hnpark@heraldcorp.com)