Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired hematopoietic stem cell clonal disease. Mutations in the PIGA gene cause the absence of complement regulatory proteins such as CD55 and CD59, which are the main molecular basis for complement-mediated intravascular hemolysis in PNH. The main clinical manifestations in PNH patients include varying degrees of paroxysmal intravascular hemolysis, paroxysmal hemoglobinuria, bone marrow hematopoietic failure, and venous thrombosis. Chronic hemolysis can cause various clinical symptoms such as fatigue, thrombosis, renal insufficiency, pain, impaired consciousness, and dyspnea. Studies show that the prognosis of PNH patients is poor before the use of complement inhibitors.

Traditional PNH treatment mainly includes symptomatic support therapy, thrombosis prevention and control, and allogeneic hematopoietic stem cell transplantation; for patients with bone marrow failure, immunosuppressive therapy can be used. According to the “Chinese Guidelines for the Diagnosis and Treatment of Paroxysmal Sleep Hypoglobinemia (2024 edition)”, PNH treatment should be based on clinical classification, and the first-line treatment for classic PNH is complement inhibitors.

Succinate pepcapan tablets (Youkupan, development code: HSK39297) are a Class 1 innovative drug independently developed by Haisco Pharmaceutical Group. They are used to treat adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have not received complement inhibitor treatment before. As a highly selective small molecule inhibitor of complement factor B (FB) with proprietary intellectual property rights, succinate pepcapan tablets are administered orally once daily. No dose adjustment is required for patients with mild or moderate renal impairment or mild to moderate liver dysfunction. There is also no need to adjust the dose when used in combination with potent CYP2C8 inhibitors. Additionally, the medication does not depend on food intake, enhancing the convenience and adherence of patient treatment.

The pantoprazole-induced clinical benefits in PNH show a triple improvement—a head-to-head Phase III study in China shows a 59.5% rate of Hb achievement, a 48.35 g/L increase in Hb, and reduced blood transfusion needs; 94.6% of patients were free from blood transfusions, saving medical resources; in terms of safety, the incidence of TEAEs is lower, and no adverse events leading to drug withdrawal or trial withdrawal occurred; in terms of convenience, it can be taken once daily orally, with a steady-state half-life (t1/2) of approximately 25 hours.

Hisicor is an international pharmaceutical company integrating research and development, production, and sales (stock code 002653.SZ). It focuses on areas such as anesthesia and analgesia, chronic disease metabolism, digestive liver diseases, respiratory autoimmune diseases, the nervous system, and tumors. Adhering to innovation and globalization strategies, with the vision of “becoming the most trusted international pharmaceutical company,” Hisicor aims to meet unmet clinical needs and care for patients worldwide. Currently, Hisicor Pharmaceutical Group has established a range of diversified research and development programs with independent intellectual property rights, covering small molecule innovative drugs, biopharmaceuticals, and high-end generic drugs. Many projects are in the clinical development stage, and several innovative drugs have been commercialized, providing diverse innovative product solutions to the world.

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