FDA接受明星公司Loxo的明星药物Larotrectinib的上市申请，并授予优先审评资格！

最新消息！

美国Loxo肿瘤学（Loxo Oncology）公司是一家知名度与日俱增的生物制药公司，是2017 ASCO大会上冉冉升起的一颗明星，旨在为患有遗传缺陷的癌症患者开发高选择性创新药物。该公司于2018年5月29日宣布，FDA已经接受了其在研药物larotrectinib的新药申请（NDA）并授予了优先审评资格，该药物用于治疗患有NTRK基因融合的晚期或转移性实体瘤的成人和儿童患者。根据“处方药使用者费用法”（PDUFA），FDA已经将目标批准日期设定为2018年11月26日。

Loxo Oncology首席执行官Josh Bilenker博士表示：“我们很高兴larotrectinib的NDA已被FDA接受并获得优先审评资格。Larotrectinib标志着治疗癌症方面的一个重要转变，癌症治疗将基于肿瘤的遗传学特征，而不再是癌症在体内的起源部位。”

除了优先审评资格之外，Larotrectinib也获得了FDA授予的突破性疗法认定、罕见儿科疾病认定和孤儿药资格。

Loxo公司和拜耳公司正在开展larotrectinib的开发和商业化合作。拜耳计划于2018年向欧盟提交上市申请（MAA）。

如果获批，Larotrectinib将成为首款不区分肿瘤来源的小分子抗癌药物，具有划时代意义。2017年5月23日，美国FDA传来一条重量级消息——由默沙东（MSD）带来的KEYTRUDA（pembrolizumab）获批治疗带有微卫星不稳定性高（microsatellite instability-high，MSI-H）或错配修复缺陷（mismatch repair deficient，dMMR）的实体瘤患者，他们的病情在先前的治疗后都出现了进展。值得一提的是，这是美国FDA批准的首款不依照肿瘤来源，而是依照生物标志物进行区分的抗肿瘤疗法，具有里程碑式的意义。

请阅读新闻稿了解详情。药时代将继续密切关注，及时报道。

FDA Accepts Larotrectinib New Drug Application and Grants Priority Review

GlobeNewswire May 29, 2018

– PDUFA date set for November 26, 2018 –

STAMFORD, Conn., May 29, 2018 (GLOBE NEWSWIRE) -- Loxo Oncology, Inc. (LOXO), a biopharmaceutical company innovating the development of highly selective medicines for patients with genetically defined cancers, today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) and granted Priority Review for larotrectinib for the treatment of adult and pediatric patients with locally advanced or metastatic solid tumors harboring an NTRK gene fusion. The FDA has set a target action date of November 26, 2018, under the Prescription Drug User Fee Act (PDUFA).  

“We are excited the larotrectinib NDA has been accepted by FDA and granted Priority Review status,” said Josh Bilenker, M.D., chief executive officer of Loxo Oncology. “Larotrectinib marks an important shift towards treating cancer based on the tumor’s genetics rather than its site of origin in the body.”

The FDA grants Priority Review for the applications of medicines that, if approved, would provide significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. Larotrectinib has also been granted Breakthrough Therapy Designation, Rare Pediatric Disease Designation and Orphan Drug Designation by the FDA.

Loxo Oncology and Bayer are engaged in a collaboration for the development and commercialization of larotrectinib. Bayer plans to submit a Marketing Authorization Application (MAA) in the European Union in 2018.

About Larotrectinib (LOXO-101)
Larotrectinib is an oral and highly selective investigational tropomyosin receptor kinase (TRK) inhibitor in clinical development for the treatment of patients with cancers that harbor a neurotrophic tyrosine receptor kinase (NTRK) gene fusion. Growing research suggests that the NTRK genes, which encode for TRKs, can become abnormally fused to other genes, resulting in growth signals that can lead to cancer in many sites of the body. In clinical trials, larotrectinib demonstrated anti-tumor activity in patients with tumors harboring NTRK gene fusions, regardless of patient age or tumor type. In an analysis of 55 RECIST-evaluable adult and pediatric patients with NTRK gene fusions, larotrectinib demonstrated a 75 percent centrally-assessed confirmed overall response rate (ORR) and an 80 percent investigator-assessed confirmed ORR, across many different types of solid tumors. The majority of all adverse events were grade 1 or 2.

Larotrectinib has been granted Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation and Orphan Drug Designation by the U.S. FDA.

In November 2017, Loxo Oncology and Bayer entered into an exclusive global collaboration for the development and commercialization of larotrectinib and LOXO-195, a next-generation TRK inhibitor. Bayer and Loxo Oncology will jointly develop the two products with Loxo Oncology leading the ongoing clinical studies as well as the filing in the U.S., and Bayer leading ex-U.S. regulatory activities and worldwide commercial activities. In the U.S., Loxo Oncology and Bayer will co-promote the products.

For additional information about the larotrectinib clinical trials, please refer to www.clinicaltrials.gov. Interested patients and physicians can contact the Loxo Oncology Physician and Patient Clinical Trial Hotline at 1-855-NTRK-123 or visit www.loxooncologytrials.com/trk-trials.

About TRK Fusion Cancer
TRK fusion cancer occurs when a neurotrophic tyrosine receptor kinase (NTRK) gene fuses with another unrelated gene, producing an altered tropomyosin receptor kinase (TRK) protein. The altered protein, or TRK fusion protein, is constantly active, triggering a permanent signal cascade. These proteins become the primary driver of the spread and growth of tumors in patients with TRK fusion cancer. TRK fusion cancer is not limited to certain types of cells or tissues and can occur in any part of the body. NTRK gene fusions occur in various adult and pediatric solid tumors with varying prevalence, including appendiceal cancer, breast cancer, cholangiocarcinoma, colorectal cancer, GIST, infantile fibrosarcoma, lung cancer, mammary analogue secretory carcinoma of the salivary gland, melanoma, pancreatic cancer, thyroid cancer, and various sarcomas. It may affect greater than 60 percent of both adult and pediatric patients with certain rare tumor types, such as secretory breast, secretory salivary gland and infantile fibrosarcoma. Only sensitive and specific tests can reliably detect TRK fusion cancer. Next-generation sequencing (NGS) can provide a comprehensive view of genomic alterations across a large number of genes. Fluorescence in situ hybridization (FISH) can also be used to test for TRK fusion cancer, and immunohistochemistry (IHC) can be used to detect the presence of TRK protein

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