FDA approves LONSURF for treatment of patients with metastatic colorectal cancer

Taiho Oncology, Inc. (U.S.), a subsidiary of Taiho Pharmaceutical Co., Ltd. (Japan), today announced that the U.S. Food and Drug Administration (FDA) approved LONSURF^® (trifluridine and tipiracil), formerly known as TAS-102, for the treatment of patients with metastatic colorectal cancer (mCRC) who have been previously treated with fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy, an anti-VEGF biological therapy, and if RAS wild-type, an anti-EGFR therapy.

"Patients with metastatic colorectal cancer, whose disease has progressed after treatment with standard therapies, have had limited therapeutic options to treat their disease," said Eric Benn, Taiho Oncology's President and Chief Executive Officer. "LONSURF helps address this unmet medical need by providing patients with a new therapeutic option that can help extend their overall survival. As the first FDA approval for Taiho Oncology, LONSURF also represents a major milestone for our company."

"Metastatic colorectal cancer cells often become resistant to previously effective treatment, underscoring the importance of identifying new therapeutic options for patients with the disease," said Robert J. Mayer, MD, Faculty Vice President for Academic Affairs at the Dana Farber Cancer Institute, Professor of Medicine at Harvard Medical School, and Principal Investigator of the RECOURSE study. "In a pivotal clinical trial, LONSURF demonstrated that it can extend overall survival, providing patients and their oncologists with a novel oral therapy."

Pivotal Phase III Study Demonstrates Improvement in Overall Survival (OS)
The FDA approval of LONSURF is based on results from the global Phase III RECOURSE trial in 800 patients who have been previously treated for mCRC.¹

The trial met the primary efficacy endpoint of statistically significant improvement in OS compared to placebo (HR = 0.68, CI: 0.58, 0.81, p < 0.001). LONSURF reduced the risk of death by 32 percent when compared to placebo. Median OS was 7.1 months (95 percent CI: 6.5, 7.8) and 5.3 months (95 percent CI: 4.6, 6.0) for LONSURF and placebo, respectively.

LONSURF caused severe and life-threatening myelosuppression (Grade 3-4) consisting of anemia (18%), neutropenia (38%), thrombocytopenia (5%), and febrile neutropenia (3.8%). Complete blood counts should be obtained prior to and on Day 15 of each cycle of LONSURF and more frequently as clinically indicated. One patient (0.2%) died due to neutropenic infection.

The most common adverse drug reactions or laboratory abnormalities were anemia, neutropenia, asthenia/fatigue, nausea, thrombocytopenia, decreased appetite, diarrhea, vomiting, abdominal pain, and pyrexia. The most common adverse reactions leading to dose reduction were neutropenia, anemia, febrile neutropenia, fatigue, and diarrhea.¹

"Data from the pivotal RECOURSE study demonstrated a significant survival benefit for patients with refractory metastatic colorectal cancer, whose disease had progressed after standard therapies, reducing the risk of death in these patients by 32 percent," said Fabio Benedetti, MD, Taiho Oncology's Senior Vice President and Chief Medical Officer. "This significant milestone underscores our longstanding commitment to patients challenged by metastatic colorectal cancer and to bringing new treatment options to them."

LONSURF was approved in Japan in March 2014 and is indicated to treat unresectable advanced or recurrent colorectal cancer.