FDA批准口服广谱抗癌药针对17种带有NTRK基因融合的肿瘤

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中文报导

http://www.wenxuecity.com/news/2018/11/27/7857215.html

https://mp.weixin.qq.com/s/34vIrozubBtMnqOzAHxBkw

美国上市时间：2018年11月26日

批准单位：美国食品和药物管理局（FDA）

药物名称：Vitrakvi（又名Larotrectinib）

药物功效：有效对抗由单一罕见基因突变驱动的各种癌症

针对群体：患有实体肿瘤的成人和儿童患者

制造商：Bayer（拜耳）和Loxo Oncology共同研发

可有效治疗的类型：

肺癌、甲状腺癌、黑色素瘤

胃肠癌、结肠癌、软组织肉瘤

唾液腺、婴儿纤维肉瘤、阑尾癌

乳腺癌、胆管癌、胰腺癌等17种

中文版：美FDA批准首个TRK抑制剂治疗NTRK基因融合晚期实体瘤

http://med.sina.cn/article_detail_103_2_56652.html#

FDA官方公布：FDA approves larotrectinib for solid tumors with NTRK gene fusions

https://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm626720.htm

On November 26, 2018, the Food and Drug Administration granted accelerated approval to larotrectinib (VITRAKVI, Loxo Oncology Inc. and Bayer) for adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation, that are either metastatic or where surgical resection is likely to result in severe morbidity, and who have no satisfactory alternative treatments or whose cancer has progressed following treatment.

This is the second tissue-agnostic FDA approval for the treatment of cancer.

Approval was based on data from three multicenter, open-label, single-arm clinical trials: LOXO-TRK-14001 (NCT02122913), SCOUT (NCT02637687), and NAVIGATE (NCT02576431). Identification of positive NTRK gene fusion status was prospectively determined in local laboratories using next generation sequencing (NGS) or fluorescence in situ hybridization (FISH). NTRK gene fusions were inferred in three pediatric patients with infantile fibrosarcoma who had a documented ETV6 translocation by FISH. The major efficacy outcome measures were overall response rate (ORR) and response duration, as determined by a blinded independent review committee according to RECIST 1.1.

Efficacy was evaluated in the first 55 patients with unresectable or metastatic solid tumors harboring an NTRK gene fusion enrolled across the three trials. All patients were required to have progressed following systemic therapy for their disease, if available, or would have required surgery with significant morbidity for locally advanced disease. Twelve patients were less than 18 years of age. A total of 12 cancer types were represented, with the most common being salivary gland tumors (22%), soft tissue sarcoma (20%), infantile fibrosarcoma (13%), and thyroid cancer (9%).

ORR was 75% (95% CI: 61%, 85%), including 22% complete responses and 53% partial responses. At the time of database lock, median duration of response had not been reached. Response duration was 6 months or longer for 73%, 9 months or longer for 63%, and 12 months or longer for 39% of patients.

The safety of larotrectinib was evaluated in 176 patients enrolled across the three clinical trials, including 44 pediatric patients. The most common adverse reactions (≥20%) with larotrectinib were fatigue, nausea, dizziness, vomiting, increased AST, cough, increased ALT, constipation, and diarrhea.

The recommended larotrectinib doses are 100 mg orally twice daily for adults and 100 mg/m2 orally twice daily (maximum of 100 mg per dose) for pediatric patients.

View full prescribing information for VITRAKVI.

This indication is approved under accelerated approval and continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. FDA granted this application priority review, breakthrough therapy designation and orphan product designation. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.