▎WuXi AppTec Content Team Report

NMOSD isA rare and debilitating autoimmune disease of the central nervous system, mainly involving the optic nerve and spinal cord. Most NMOSD patients will experience multiple relapses, with possible new neurological symptoms or worsening of existing neurological symptoms, leading to permanent disability due to repeated relapses.

Eculizumab is a C5 complement inhibitor that works by selectively inhibiting the activation of terminal complement C5.. The complement system is an important part of the human immune system, but when the complement is activated in an uncontrolled manner, it may lead to the body attacking its own healthy cells. Eculizumab is administered intravenously every two weeks after the induction dose period. Previously, the drug has been approved for use in China for the treatment of adults and children.Paroxysmal Nocturnal Hemoglobinuria (PNH)、Atypical Hemolytic Uremic Syndrome (aHUS)And adult anti-acetylcholine receptor (AChR) antibody-positive refractoryGeneralized Myasthenia Gravis (gMG)。

Eculizumab Approved in ChinaNMOSD Indications，Mainly based on the results of a pivotal Phase 3 PREVENT studyThe study results showed that, based on the time to first adjudicated relapse during the trial period, eculizumab treatment significantly reduced the risk of relapse with both statistical and clinical significance.At week 48, 98% of patients treated with eculizumab (63% of placebo-treated patients) were relapse-free (relative risk reduction of 94.2%), and the treatment benefit persisted until week 144.. In addition,In the 144-week PREVENT study, 96% of patients treated with eculizumab were relapse-free, compared to only 45% in the placebo group.The safety and tolerability of eculizumab were consistent during the PREVENT study and its open-label extension study. The most common adverse reactions during treatment were upper respiratory tract infections, headaches, nasopharyngitis, and nausea.

Chief Physician of the Department of Neurology at Peking Union Medical College Hospital, Deputy Head of the Neuroimmunology Group of the Neurology Branch of the Chinese Medical AssociationProfessor Xu YanSaid: "The high recurrence rate of NMOSD often leads to long-term severe disability in patients. Therefore, we are very pleased to see that the Phase 3 PREVENT study has confirmed the safety and efficacy of the C5 complement inhibitor in reducing NMOSD relapses.Almost all patients treated with eculizumab did not relapse at 48 weeks. The approval of the indication for eculizumab in the treatment of adult NMOSD in China is of great significance to the treatment in this field in our country."

CEO of AstraZeneca Rare Diseases Subsidiary Raison PharmaceuticalsMr. Marc Dunoyer"Noted: 'Patients with NMOSD and their families should not live in fear of relapses and potential complications. Eculizumab is the world's first C5 complement inhibitor for the treatment of NMOSD, and we are delighted to introduce it in China, providing more innovative treatment options for patients with NMOSD. The approval of eculizumab further enhances its accessibility in the global market and fully demonstrates our commitment to improving the quality of life for patients with rare neurological disorders.'"

Executive Vice President of AstraZeneca Global, President of International Business and ChinaMr. Wang LeiSaid: "Providing patients with high-quality medical services is our unwavering初心 (original aspiration), and we look forward to eculizumab bringing long-term survival benefits to NMOSD patients. In the future, we will continue to comprehensively and rapidly introduce innovative global medicines. We will also work with like-minded partners to build a robust rare disease diagnosis and treatment ecosystem, striving to create a better future for the NMOSD community and the broader rare disease population."