Recently, AstraZeneca’s rare disease drug Vonjo (generic name: pacritinib) has been approved for marketing in China by the National Medical Products Administration (NMPA). The drug is indicated for adult patients with myelofibrosis (MF) and severe thrombocytopenia. Myelofibrosis is a rare myeloproliferative neoplasm that often leads to anemia, splenomegaly, and systemic symptoms. Treatment options for patients with platelet counts below 50×10⁹/L have been extremely limited. As an oral JAK2/IRAK1 inhibitor, Vonjo effectively reduces spleen size and alleviates disease-related symptoms while demonstrating a favorable safety profile in thrombocytopenic patients. The approval was supported by data from the pivotal Phase III PERSIST-2 clinical trial, which showed that Vonjo significantly improved spleen volume reduction and symptom burden compared to existing therapies. As a key part of AstraZeneca’s strategy in rare diseases, the launch of Vonjo offers new therapeutic hope for Chinese patients with rare hematologic disorders and addresses a critical unmet medical need.
近日，阿斯利康（AstraZeneca）旗下罕见病新药万诺维（Vonjo，通用名：pacritinib）在中国获得国家药品监督管理局（NMPA）批准上市。该药物主要用于治疗伴有严重血小板减少症的骨髓纤维化（Myelofibrosis, MF）成人患者。骨髓纤维化是一种罕见的骨髓增殖性肿瘤，常导致贫血、脾肿大及全身症状，而血小板计数低于50×10⁹/L的患者治疗选择极为有限。万诺维作为一种口服JAK2/IRAK1抑制剂，能有效缓解脾脏肿大和疾病相关症状，同时对低血小板患者具有良好的安全性。此次获批基于关键III期临床试验PERSIST-2的数据，结果显示，与现有疗法相比，万诺维显著改善了患者的脾脏体积和症状负担。作为阿斯利康在罕见病领域的重要布局之一，万诺维的上市将为中国罕见血液病患者提供新的治疗希望，并填补当前临床治疗空白。
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