▎WuXiEdited by Kant Content Team

On June 22, the U.S. FDA website showed that Roche's newly developed C5 cyclic antibody Piasky (crovalimab,Covalemonab) has been approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).) Patient.According to previous press releases, Piasky is the first monthly subcutaneous treatment for PNH, and patients have the option to self-administer the drug outside of supervised medical facilities.

PNH is a rare and life-threatening syndrome, with patients suddenly experiencing symptoms such as hematuria, anemia, and thrombosis.PNH patients have significantly higher levels of lactate dehydrogenase (LDH) than normal, and LDH is a marker of hemolysis. Hemolysis increases the risk of thrombosis, which is the main cause of death in PNH patients.Inhibition of complement C5 is the standard treatment for PNH patients with significant clinical symptoms.However, the continuous complete suppression of the terminal complement pathway and the high serum concentration of complement C5 pose challenges for drug development, leaving patients with only intravenous treatment options.

According to data presented at the American Society of Hematology (ASH) Annual Meeting in December 2022,Covalemonab inIn the COMMODORE 3 study, the co-primary efficacy endpoints were met – the proportion of subjects with hemolysis control as measured by LDH levels, and the proportion of subjects avoiding transfusion (TA).

Specifically,From Week 5 to Week 25, the average proportion of subjects with controlled hemolysis was 78.7%. The difference between the proportion of subjects achieving TA within 24 weeks before screening (0.0%) and the proportion achieving TA from baseline to Week 25 (51.0%) was statistically significant.It is worth mentioning that the demand for blood transfusion is an important clinical indicator of hemolysis caused by PNH complement disorder.

According to reports, during useCovalemonabWithin two weeks after treatment, the fatigue status of the subjects showed rapid and clinically significant improvement, which continued over time. The overall safety data in the trial were consistent with the known safety profile of C5 inhibitors and the underlying disease, indicating...KefaliteMonoclonal AntibodyWell tolerated, with no new safety signals identified.

Covalemonab is a recyclable complement protein C5-targetedHumanizedMonoclonal antibody designed to block the complement system in the human immune system. The product has been engineered through continuous monoclonal antibody recovery technology.Binding to C5 can induce the degradation of the C5 protein, after which this antibody can be released back into the extracellular space by binding to the FcRn receptor, allowing it to bind to other C5 proteins.Therefore, Cofavelimab can achieve rapid and sustained complement pathway inhibition at a lower dose.

According to an earlier press release from Roche, the efficacy and convenience of Covarimab in treating PNH have been significantly improved. In conventional blood transfusion therapy, PNH patients need to be hospitalized repeatedly for blood transfusions due to ongoing hemolysis, with each transfusion typically taking 3-4 hours.Covale单抗可每4周1次自行皮下注射，便利性更高。In February this year,China's National Medical Products Administration (NMPA) approved the therapyListed，For adult and adolescent (≥12 years) patients with paroxysmal nocturnal hemoglobinuria who have not received complement inhibitor therapy.

Currently, there are several PNH therapies in clinical stages of research. It is hoped that these therapies under development can be as promising as today's.Just like Covralimab, it was finally approved successfully, benefiting more PNH patients.

▲ As of June 17, 2024, globally activeR&DClinical Status of PNH Therapy Review

[2] FDA Accepts Application for Genentech’s Crovalimab for the Treatment of PNH, a Rare Life-Threatening Blood Condition. Retrieved June 21, 2024 from https://www.gene.com/media/press-releases/15000/2023-09-04/fda-accepts-application-for-genentechs-c

[3] The National Medical Products Administration Approves the Marketing of Corvalimab Injection. RetrievedJune 21, 2024, from https://www.nmpa.gov.cn/zhuanti/cxylqx/cxypxx/20240207173310135.html