▎WuXiEdited by Kant Content Team

Recently, Roche announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion on its next-generation C5 recycling antibody Piasky (crovalimab) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). The CHMP recommends PiaSky for use in adults and adolescents (12 years of age and older with a body weight of no less than 40 kg) who have either not been previously treated or have already been treated with a C5 inhibitor.If approved, PiaSky will become the first monthly subcutaneous (SC) injection PNH therapy in the EU, and patients can self-administer after sufficient training.This therapy may become an alternative to the current regular intravenous infusion of C5 inhibitor therapy, with the potential to help reduce the burden and life disruption for PNH patients and their caregivers.

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, which is a marker of hemolysis. Hemolysis increases the risk of thrombosis, a leading 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.

The CHMP recommendation is primarily based on the results of the Phase 3 COMMODORE2 study, which involved PNH patients who had not received prior C5 inhibitor treatment. The study results showed,PiaSky, administered subcutaneously once every 4 weeks, demonstrated good disease control and tolerability. The efficacy of PiaSky was non-inferior to the standard therapy, eculizumab, a C5 inhibitor that requires intravenous infusion once every 2 weeks.The incidence of adverse events in patients receiving PiaSky treatment was similar to that of the active comparator drug (78% and 80%, respectively). The application also includes supportive data from two additional Phase 3 studies: the COMMODORE 1 study (in PNH patients switching from currently approved C5 inhibitor therapies) and the COMMODORE 3 study (in Chinese patients newly receiving C5 inhibitor treatment).

PiaskyIt is a recyclable product targeting complement protein C5.HumanizedMonoclonal 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,PiaskyRapid and sustained complement pathway inhibition can be achieved at lower doses.

According to an earlier press release from Roche,PiaskyThe effectiveness and convenience of PHN treatment have greatly improved. In conventional blood transfusion therapy, PNH patients need to be hospitalized repeatedly due to ongoing hemolysis, and each transfusion session typically lasts 3-4 hours.PiaskyCan be self-administered subcutaneously once every 4 weeks, offering higher convenience.In February this year,Approved by the National Medical Products Administration (NMPA) of ChinaListed，For adult and adolescent (≥12 years) PNH patients who have not received complement inhibitor therapy. This month, the U.S. FDAApprovalPiasky LaunchUsed to treat patients with PNH.

Currently, there are several PNH therapies in clinical stages of development. We look forward to these therapies in development.Ultimately, it was successfully approved, benefiting more PNH patients.

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

Note: This table was compiled by the WuXi AppTec content team based on publicly available information and is not a comprehensive list. If there are any omissions, please feel free to add them.

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References:

[1] CHMP recommends EU approval of Roche’s PiaSky for people with PNH, a rare, life-threatening blood condition. Retrieved June 28, 2024 fromhttps://www.globenewswire.com/news-release/2024/06/28/2905778/0/en/CHMP-recommends-EU-approval-of-Roche-s-PiaSky-for-people-with-PNH-a-rare-life-threatening-blood-condition.html

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