9月14日，FDA加速批准拜耳Aliqopa（copanlisib）用于治疗复发性滤泡性淋巴瘤，适用于既往至少接受过两种系统性治疗的成人患者。
FDA药物评价与研究中心血液学和肿瘤产品办公室代理主任兼FDA肿瘤优化中心主任Pazdur称：对于复发性滤泡性淋巴瘤患者来说，这种癌症即使在多种治疗后也会复发。这些患者的选择是有限的，今天的批准为他们提供了额外的治疗选择，填补了他们未满足的需求。
滤泡性淋巴瘤是一种生长缓慢的非霍奇金淋巴瘤，是淋巴系统的一种癌症。淋巴系统是人体免疫系统的一部分，由淋巴组织、淋巴结、脾、胸腺、扁桃体及骨髓组成。美国国立卫生研究院的国家癌症研究所估计，今年美国约有 72240人将被诊断患有某种形式的非霍奇金淋巴瘤，大约20140名非霍奇金淋巴瘤患者将在2017年死于这种疾病。
Aliqopa是一种激酶抑制剂，它通过阻止几种促使细胞生长的酶起作用。这次Aliqopa是通过加速批准而获批，这种批准通道允许FDA采用被认为可预测患者临床获益的临床试验数据来批准用于严重疾病的药物，以填补一种未满足的医疗需求。Aliqopa获批后，还需要进行进一步的临床试验，以证实该药物的临床获益，拜耳目前正在进行这些研究。
今天对Aliqopa的批准是基于一项单组试验的数据，该试验包括了104名滤泡性B细胞非霍奇金淋巴瘤患者，这些患者在既往至少接受过两种治疗后疾病出现复发。该试验检测了治疗后肿瘤经历完全或部分缩小的患者人数（总缓解率）。试验中，59%的患者有中值12.2个月的完全或部分缓解。
Aliqopa的常见副作用包括高血糖、腹泻、一般性力量和能量下降、高血压、某些白血球水平减少 (白血球减少症，嗜中性白血球减少症)、恶心、下呼吸道感染、血小板计数减少（血小板减少症）。严重副作用包括感染、高血糖、高血压、肺部组织炎症（非传染性肺炎）、某些白血球减少（嗜中性白血球减少症）和严重的皮肤反应。孕妇或哺乳期妇女不应服用Aliqopa，因为它可能对发育中的胎儿或新生儿造成伤害。
Aliqopa被授予了优先审评资格，基于这一资格，FDA要在6个月内对一款药物做出是否批准的决定，如果获得批准，该药物将显著改善治疗、诊断或预防严重疾病的安全性或有效性。Aliqopa还获得了孤儿药资格，这一资格可提供激励措施，以帮助和鼓励用于罕见疾病药物的开发。
Bayer Receives FDA Approval for Aliqopa™ (copanlisib) 60 mg vial for Injection in Adults with Relapsed Follicular Lymphoma after Two Prior Systemic Therapies
- Accelerated approval based on overall response rate (ORR) of 104 adult patients with relapsed follicular lymphoma (FL) from the Phase II CHRONOS-1 study
- Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial
- Aliqopa achieved a 59% ORR in patients with relapsed FL [n=104 (95% CI 49, 68)]
- First approval of an intravenous phosphatidylinositol-3-kinase (PI3K) inhibitor
WHIPPANY, N.J., Sept. 14, 2017 /PRNewswire/ -- Bayer announced today that the U.S. Food and Drug Administration (FDA) has approved Aliqopa™ (copanlisib) for the treatment of adult patients with relapsed FL who have received at least two prior systemic therapies.1 Accelerated approval was granted for this indication based on ORR. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
Aliqopa is a novel intravenous PI3K inhibitor with inhibitory activity predominantly against the PI3K-alpha and PI3K-delta isoforms expressed in malignant B cells.1 The FDA granted approval under the accelerated approval pathway based on data from the open-label, single-arm Phase II CHRONOS-1 [NCT01660451] trial investigating Aliqopa in 104 adult patients with follicular B-cell non-Hodgkin's lymphoma (NHL) who had relapsed disease following at least two prior systemic therapies.
FL is the most common subtype of indolent, or slow-growing, NHL, comprising approximately one out of five lymphomas in the U.S.2,3 Response rates and duration of response decline with each line of therapy, underscoring the need for patients whose disease has already progressed.
"Before the approval of Aliqopa, doctors and patients facing follicular lymphoma, a very serious and difficult-to-treat blood cancer, have had a need for new and effective options when patients relapse," said Carsten Brunn, Head of Bayer Pharmaceuticals, Americas Region. "The FDA's accelerated approval of this important treatment reflects Bayer's commitment to delivering new treatment options, and we commend the FDA on its speedy review and ongoin |
