Objective

Contemporary management of patients with neuro-oncologic disease requires an understanding of approvals by the US Food and Drug Administration (FDA) related to nervous system tumors. To summarize FDA updates applicable to neuro-oncology practitioners, we sought to review oncology product approvals and Guidances that were pertinent to the field in the past year.

Methods

Oncology product approvals between January 1, 2020, and December 31, 2020, were reviewed for clinical trial outcomes involving tumors of the nervous system. FDA Guidances relevant to neuro-oncology were also reviewed.

Results

Five oncology product approvals described outcomes for nervous system tumors in the year 2020. These included the first regulatory approval for neurofibromatosis type 1: selumetinib for children with symptomatic, inoperable plexiform neurofibromas. Additionally, there were 4 regulatory approvals for non-central nervous system (CNS) cancers that described clinical outcomes for patients with brain metastases. These included the approval of tucatinib for metastatic human epidermal growth factor receptor 2 (HER2)-positive breast cancer including patients with brain metastases, brigatinib for anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC), and pralsetinib and selpercatinib for RET fusion-positive NSCLC. Finally, two FDA Guidances for Industry, “Cancer Clinical Trial Eligibility Criteria: Brain Metastases” and “Evaluating Cancer Drugs in Patients with Central Nervous System Metastases” were published to facilitate drug development for and inclusion of patients with CNS metastases in clinical trials.

Conclusions

Despite the challenges of the past year brought on by the COVID-19 pandemic, progress continues to be made in neuro-oncology. These include first-of-their-kind FDA approvals and Guidances that are relevant to the management of patients with nervous system tumors.

中文翻译：

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美国食品和药物管理局在神经肿瘤学方面的监管更新

客观的

神经肿瘤疾病患者的现代管理需要了解美国食品和药物管理局 (FDA) 与神经系统肿瘤相关的批准情况。为了总结适用于神经肿瘤从业者的 FDA 更新，我们试图审查过去一年与该领域相关的肿瘤产品批准和指南。

方法

对 2020 年 1 月 1 日至 2020 年 12 月 31 日期间的肿瘤产品批准进行了审查，以评估涉及神经系统肿瘤的临床试验结果。还审查了与神经肿瘤学相关的 FDA 指南。

结果

五项肿瘤产品批准描述了 2020 年神经系统肿瘤的结果。其中包括第一个监管批准 1 型神经纤维瘤病：selumetinib 用于有症状、不可手术的丛状神经纤维瘤儿童。此外，还有 4 项非中枢神经系统 (CNS) 癌症的监管批准描述了脑转移患者的临床结果。其中包括批准 tucatinib 用于转移性人表皮生长因子受体 2 (HER2) 阳性乳腺癌，包括脑转移患者，brigatinib 用于间变性淋巴瘤激酶 (ALK) 阳性非小细胞肺癌 (NSCLC)，以及 pralsetinib 和用于RET的 selpercatinib融合阳性非小细胞肺癌。最后，FDA 发布了两个行业指南，“癌症临床试验资格标准：脑转移”和“评估中枢神经系统转移患者的癌症药物”，以促进 CNS 转移患者的药物开发和纳入临床试验。

结论

尽管过去一年 COVID-19 大流行带来了挑战，但神经肿瘤学仍在继续取得进展。其中包括首创的与神经系统肿瘤患者管理相关的 FDA 批准和指南。