The U.S. Food and Drug Administration has granted a rare pediatric disease (RPD) designation to Hanmi's HM15912, a drug candidate for short bowel syndrome.
|Hanmi Pharmaceutical’s HM15912 has been designated as a rare pediatric disease drug by the U.S. FDA. (Hanmi Pharmaceutical)|
Short bowel syndrome is a rare disease that causes malnutrition due to malabsorption caused by loss of more than 60 percent of the entire small intestine after congenital or postoperative resection. As the illness can be life-threatening in severe cases, patients need artificial nutritional supplements, such as total parenteral nutrition, for everyday life.
Pediatric short bowel syndrome, known to occur in about 24.5 out of 100,000 newborns, has a significant impact on the growth of children and adolescents, as the total parenteral nutrition treatment can take more than 10 hours a day.
Pediatric patients are also vulnerable to serious side effects such as sepsis and thrombosis due to infection at the injection site.
Hanmi is developing the treatment so that patients can administer it once a month based on HM15912's improved in-vivo persistence and excellent chondrocyte growth-promoting effect.
The company expects that the treatment will dramatically improve the quality of life of children with short bowel syndrome.
"Through continuous research and development and expansion of indications, the company will do its best to dramatically improve the quality of life of patients worldwide, including infants and children suffering from rare diseases," Hanmi Pharmaceutical President and CEO Kwon Se-chang said.
Hanmi is conducting a phase 1 clinical trial in Korea for the treatment and plans to begin phase 2 clinical trials in the U.S. and Europe by the second half of this year.
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