Can eculizumab cure pnh?

Eculizumab (Eculizumab) is a drug used to treat paroxysmal nocturnal hemoglobinuria (PNH). It can effectively control and reduce hemolytic attacks in PNH patients and improve related symptoms. Although eculizumab cannot completely cure PNH, it can help patients maintain a better quality of life and prolong survival.

Paroxysmal nocturnal hemoglobinuria is caused by somatic mutations in the phosphatidylinositol glycanA (PIG-A) gene on hematopoietic stem cells. The cause is complex and the possibility of complete cure is relatively small. The treatment options are mainly supportive, including blood transfusion, erythropoiesis-stimulating agents, corticosteroids, anabolic steroids, iron therapy, thromboprophylaxis, and sometimes thrombolytic therapy or allogeneic bone marrow transplantation. Eculizumab was first introduced in 2004 in an initial pilot study of 11 participants and was found to significantly reduce intravascular hemolysis and the need for blood transfusions. Two phase III trials, TRIUMPH and SHEPHERD, demonstrated that eculizumab in PNH is effective, safe, and well-tolerated in a standardized setting with predefined outcome criteria.

The generic drug Eculizumab has been launched in China and is covered by medical insurance. It is only available for reimbursement to eligible patients. The specification is 300mg (30ml) and the price per box may be more than 3,000 yuan. It is understood that the price of each box of the original Eculizumab drug sold overseas is more than 6,000 US dollars (the price may fluctuate due to the exchange rate). The ingredients of the original drug sold at home and abroad are basically the same, and the price of the original drug is still relatively expensive. There are currently no generic versions of eculizumab available on the market.