[바이오 키워드] Paroxysmal nocturnal hemoglobinuria
Enter 2023.07.10 11:23 Modify 2023.07.10 11:24 Hits 145 Enter 2023.07.10 11:23 Modify 2023.07.10 11:24 Hits 145
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Paroxysmal nocturnal hemoglobinuria (PNH), a rare disease, is a rare blood disease affecting 15.9 per 1 million population. It occurs when abnormal red blood cells produced by an acquired mutation in the PIGA gene, an enzyme involved in the synthesis of cell surface proteins, are destroyed by unregulated complement activation.
Fatigue (96%), anemia (88%), and shortness of breath (66%) are commonly observed as major symptoms, and hemolysis of red blood cells may be accompanied by a peculiar symptom of cola-colored urine at night. If untreated, 2 to 4 out of 10 people may die within 5 to 6 years after diagnosis, so timely diagnosis and treatment are necessary because it is a severe disease.
Treatment is to administer steroids for the purpose of relieving symptoms, but it is known that most patients do not show significant improvement. In this situation, an orphan drug called Ultomiris (ingredient name: rabulizumab), which inhibits the complement activation mechanism, entered prescription rights.
Ultomiris is a treatment developed by Alexion of the United States and has been sold by AstraZeneca Korea since February of this year after Alexion was merged with AstraZeneca. It was launched in the US, Germany, UK, and Switzerland, and was approved in Korea in May 2020 as a treatment for paroxysmal nocturnal hemoglobinuria.
The biggest feature of Ultomiris is that the administration interval is longer than that of the existing ‘Eculizumab (product name Soliris)’. It has a half-life about 4 times longer than eculizumab, so it is possible to administer once every 8 weeks instead of once every 2 weeks. This treatment is mainly prescribed for patients with paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome (aHUS).
2023-07-10 02:32:07
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