MFDS Approves Treatment for Paroxysmal Nocturnal Hemoglobinuria

Broader Treatment Options for Pediatric Patients with Imported Orphan Drug "PiaSky Injection"

The Ministry of Food and Drug Safety announced on December 24 that it has approved the imported orphan drug "PiaSky Injection" (active ingredient: crovalimab) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adults weighing 40 kg or more and pediatric patients aged 12 years and older.

Paroxysmal nocturnal hemoglobinuria is an acquired hematopoietic stem cell disorder characterized by hemolysis, a phenomenon in which hemoglobin escapes from red blood cells, resulting in dark-colored urine at night.

PiaSky Injection suppresses complement-mediated intravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria. The complement system is a group of proteins that attack bacteria and viruses and assist immune responses.

This drug has been approved for use in pediatric patients for the first time.

The Ministry of Food and Drug Safety expressed its expectation that this approval will expand the range of treatment options for both adult and pediatric patients with paroxysmal nocturnal hemoglobinuria.