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FDA Approves First Drug for Myelofibrosis With Thrombocytopenia
Zosia Chustecka  ;   March 01, 2022

The US Food and Drug Administration (FDA) has granted accelerated approval for a new drug for the treatment of myelofibrosis, the first specifically for patients with low platelet counts.

Pacritinib (Vonjo, CTI BioPharma) is indicated for use in the treatment of adults with intermediate- or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L.

Pacritinib is a novel oral kinase inhibitor with specificity for activity against Janus associated kinase 2 (JAK2) and IRAK1, without inhibiting JAK1. The recommended dosage is 200 mg orally twice daily.  

In the United States, there are approximately 21,000 patients with myelofibrosis, notes the manufacturer. About one third develop severe thrombocytopenia.  "Myelofibrosis with severe thrombocytopenia, defined as blood platelet counts below 50 × 109/L, has been shown to result in poor survival outcomes coupled with debilitating symptoms. Limited treatment options have rendered this disease as an area of urgent unmet medical need," commented John Mascarenhas, MD, associate professor, medicine, hematology, and medical oncology, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York City.  "I am pleased to see that a new, efficacious, and safe treatment option is now available for these patients," in a company press release.

Mascarenhas was the lead investigator of the phase 3 PERSIST-2 trial that was the basis for the approval. Results from the trial were published in 2018 in JAMA Oncology and reported in detail at the time by Medscape Medical News.  Authors of an accompanying editorial noted the trial was truncated after the FDA imposed a clinical hold on pacritinib in February 2016 after reports from an earlier trial, PERSIST-1, of patient deaths related to cardiac failure and arrest as well as intracranial hemorrhage. The clinical hold was lifted in January 2017 after the manufacturer provided the FDA with more mature data.


Zosia Chustecka,  2022年3月1日

美国食品和药物管理局(FDA)加速批准了一种治疗骨髓纤维化的新药-帕克替尼(Pacritinib; Vonjo,CTI生物制药公司),这是第一种专门针对低血小板型骨髓纤维化患者的药物。


制造商指出,在美国,大约有21,000名骨髓纤维化患者。大约三分之一的人出现了严重的血小板减少症。"患有严重血小板减少症的骨髓纤维化,定义为血小板计数低于50/μL,已被证明会导致不良生存结果和衰弱的症状。纽约市西奈山医学院Tisch癌症研究所医学、血液学和医学肿瘤学副教授John Mascarenhas博士评论说:"有限的治疗方案使这种疾病成为一个急需满足的医疗需求领域" "我很高兴看到这些患者现在可以选择一种新的、有效的、安全的治疗方法"。

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