時間:2018-05-30    作者:聯創生物醫藥信息部


美國Loxo腫瘤學(Loxo Oncology)公司是一家知名度與日俱增的生物制藥公司,是2017 ASCO大會上冉冉升起的一顆明星,旨在為患有遺傳缺陷的癌癥患者開發高選擇性創新藥物。該公司于2018529日宣布,FDA已經接受了其在研藥物larotrectinib的新藥申請(NDA)并授予了優先審評資格,該藥物用于治療患有NTRK基因融合的晚期或轉移性實體瘤的成人和兒童患者。根據處方藥使用者費用法PDUFA),FDA已經將目標批準日期設定為20181126日。 


Loxo Oncology首席執行官Josh Bilenker博士表示:“我們很高興larotrectinib的NDA已被FDA接受并獲得優先審評資格。Larotrectinib標志著治療癌癥方面的一個重要轉變,癌癥治療將基于腫瘤的遺傳學特征,而不再是癌癥在體內的起源部位?!?






如果獲批,Larotrectinib將成為首款不區分腫瘤來源的小分子抗癌藥物,具有劃時代意義。2017年5月23日,美國FDA傳來一條重量級消息——由默沙東(MSD)帶來的KEYTRUDA(pembrolizumab)獲批治療帶有微衛星不穩定性高(microsatellite instability-high,MSI-H)或錯配修復缺陷(mismatch repair deficient,dMMR)的實體瘤患者,他們的病情在先前的治療后都出現了進展。值得一提的是,這是美國FDA批準的首款不依照腫瘤來源,而是依照生物標志物進行區分的抗腫瘤療法,具有里程碑式的意義。



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

—— The End ——

【版權提示】 聯創生物醫藥發布原文章與轉載文章。轉載文章的目的是推薦有價值的信息,與更多的朋友分享,我們尊重知識產權,致力于保護知識產權,盡力注明作者和出處。如果我們轉載的文章、圖片等內容存在版權等問題,敬請聯系我們0551-68596228,我們將盡全力盡快與您聯系,及時處理。衷心感謝您的理解與惠助!

更多> 聯系我們