Recently, Chengdu Shengdi Pharmaceutical Co., Ltd., a subsidiary of Hengrui Medicine, received an "Acceptance Notice" from the National Medical Products Administration. The drug application for marketing authorization of the company's independently developed Class 1 new drug, Likang Kepan Capsules of Fumarate, has been accepted. The indication is: treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who still have anemia after previous treatment with C5 complement inhibitors. This is expected to bring new options for the treatment of this rare disease.

Research on HRS-5965-302

This application for market approval is based on a multicenter, single-arm, open-label phase III clinical trial evaluating the efficacy and safety of Likang Kepan fumarate capsules (development code: HRS-5965) in PNH patients who still have anemia after 6 months of stable treatment with C5 complement inhibitors. The study was jointly led by Professor Shi Jun of the Institute of Hematology and Blood Diseases Hospital of Chinese Academy of Medical Sciences and Professor Fu Rong of Tianjin Medical University General Hospital, with 13 centers across the country participating. The primary endpoint was the proportion of subjects with hemoglobin levels ≥12 g/dL. The results showed [1] that Likang Kepan fumarate capsules were significantly effective in improving hemoglobin levels, avoiding blood transfusions, and improving fatigue.

Regarding paroxysmal nocturnal hemoglobinuria

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired hemolytic disease characterized by a deficiency of CD55 (decay-accelerating factor, DAF) and CD59 (reactive membrane lysis inhibitor, MIRL), leading to complement-mediated intravascular hemolysis. The main clinical manifestations are intravascular hemolysis, potential hematopoietic failure, and thrombosis tendency [2] . Due to its low incidence/prevalence, PNH has been included in the first batch of rare diseases in China [3] . Although existing C5 complement inhibitors can effectively control intravascular hemolysis, most patients still suffer from persistent anemia due to extravascular hemolysis [4] . In addition, C5 complement inhibitors require long-term intravenous or subcutaneous administration, and the convenience of administration needs to be improved.

Regarding Liconopyr fumarate capsules

Licancopan fumarate capsules are a Class 1 new drug independently developed by the company. It is an oral small molecule inhibitor targeting factor B in the complement activation system (AP) pathway. Factor B inhibitors can control intravascular and extravascular hemolysis by targeting factor B and inhibiting the AP pathway, thus compensating for the shortcomings of C5 complement inhibitor therapy. Furthermore, the oral administration method improves patient adherence. In addition, the marketing application for Licancopan fumarate capsules for the treatment of adult patients with pneumonephropathy (PNH) who have not previously received complement inhibitor therapy has been accepted and included in the priority review process. Simultaneously, clinical studies for its use in patients with IgA nephropathy are also underway.

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