AstraZeneca said it would introduce a highly concentrated formulation of Ultomiris (ravulizumab), which maintains the efficacy of the original formulation while reducing infusion time, in the Korean market. 

AstraZeneca Korea announced that it will launch high-concentration formulations (300mg/3mL and 1,100mg/11mL) of Ultomiris, an orphan drug approved in Korea for the treatment of the rare diseases paroxysmal nocturnal hemoglobinuria (PNH) and atypical Hemolytic Uremic Syndrome (aHUS) on July 1.

The new high-concentration formulation (100mg/mL) is 10 times more concentrated than the existing 10mg/mL formulation, aiming to minimize the medical burden on patients. The new formulation shortens intravenous infusion time by 60-70 percent with a reduced volume while maintaining the efficacy of the existing formulation.

The high-concentration Ultomiris has demonstrated similar efficacy, safety, pharmacokinetics and immunogenicity to the standard formulation for the treatment of PNH and will be covered by health insurance for this indication in Korea from July 1.

PNH is a rare hematologic disorder with an incidence of 15.9 per 1 million people and is caused by the destruction of abnormal red blood cells due to an acquired mutation in the PIGA gene by uncontrolled complement activation. Symptoms include fatigue (96 percent), anemia (88 percent), and dyspnea (66 percent). Some individuals may experience the unusual symptom of passing colored urine at night, which is attributed to the hemolysis of red blood cells.

If untreated, PNH can cause death in two to four out of 10 people within 5 to 6 years of diagnosis, so timely diagnosis and treatment are essential.