Oh Yu-Kyoung, head of the Ministery of Food and Drug Safety, speaks at a roundtable with rare disease patients and their families at the Heavy Ion Therapy Center of Severance Hospital, Yonsei Cancer Center in Sinchon, Seodaemun-gu, Seoul, on the 24th. /Courtesy of Presidential Office Press Photographers

The Ministery of Food and Drug Safety said on the 24th that it approved the rare drug Piascai injection (hereafter Piascai), used to treat paroxysmal nocturnal hemoglobinuria in children 12 and older and adults who weigh at least 40 kilograms.

Nocturnal hemoglobinuria is an acquired hematopoietic stem cell disorder in which hemoglobin escapes outside red blood cells, causing hemolysis and resulting in dark, blood-tinged urine at night.

Piascai is a treatment for paroxysmal nocturnal hemoglobinuria developed by Swiss pharmaceutical company Roche. Its treatment mechanism suppresses complement-mediated intravascular hemolysis, in which "complement," a protein that attacks bacteria and viruses and assists immune responses, is involved. The drug has now been approved for the first time for pediatric use.

The Ministery of Food and Drug Safety said it expects that "the range of treatment options for paroxysmal nocturnal hemoglobinuria in adults and children will broaden."

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