Type of medical product:
Indications for use:
Paroxysmal nocturnal hemoglobinuria
Preclinical trials in vivo
The therapeutic is a recombinant glycosylated humanized monoclonal antibody to treat paroxysmal nocturnal hemoglobinuria.
Paroxysmal nocturnal hemoglobinuria (Marchiafava–Micheli syndrome, Strubing-Marchiafava syndrome) is an acquired hemolytic anemia caused by intravascular destruction of defective erythrocytes.
Paroxysmal nocturnal hemoglobinuria (PNH) is an ultrarare disease with prevalence of 5 to 15.9 cases per million and incidence of 1.3 cases per million a year. Severe forms with prominent clinical manifestation are about 25 to 30% (1 to 5 cases per million). As to its prevalence in Russia there might be 745 to 2369 PNH patients. According to the data of the Center on Healthcare Technology Evaluation of the Russian Presidential Academy of National Economy and Public Administration in 2013 there were 215 PNH patients registered in Russia with treatment cost need of 5.3 bln. rubles.