Nexavar® (sorafenib): Applications Submitted in US and Europe for Approval; Clinical Trial Shows Positive Results on RAI-Resistant Papillary/Follicular Cancer
Update: July 1, 2013 - Applications Submitted in U.S. and Europe for Nexavar® (sorafenib) for Treatment of Radioactive Iodine-Refractory Differentiated Thyroid Cancer
July 1, 2013 — Bayer HealthCare and Onyx Pharmaceuticals have submitted applications to the U.S. Food & Drug Administration and the European Medicines Agency for approval of Nexavar® (sorafenib) for treating differentiated thyroid cancer (papillary, follicular, variants) that is locally advanced or metastatic and does not respond to radioactive iodine.
June 3, 2013 — At a plenary session at the American Society of Clinical Oncology’s Annual Meeting in Chicago, Illinois, positive results were presented from a Phase 3 Clinical Trial on the use of Nexavar® (sorafenib) tablets in thyroid cancer patients with differentiated thyroid cancer (papillary, follicular, variants) that had spread and did not respond to radioactive iodine.
The results will lead to regulatory submissions from the manufacturer to the U.S. Food and Drug Administration and to regulatory agencies globally.
The Phase 3 DECISION trial included 417 patients with locally advanced or metastatic differentiated thyroid cancer that did not respond to radioactive iodine. The trial was an international, multicenter, placebo-controlled study. The primary endpoint of the study was progression-free survival. Progression-free survival was evaluated by an independent radiological review committee, using Response Evaluation Criteria in Solid Tumors (RECIST).
Sorafenib significantly extended progression-free survival (PFS). The median PFS was 10.8 months among patients treated with sorafenib, compared to 5.8 months among patients receiving placebo. At the time of progression, patients had the option to cross over to open-label sorafenib at the discretion of the investigator. Median overall survival, a secondary endpoint of the trial, has not yet been reached in either arm of the study.
"While most patients with differentiated thyroid cancer are cured, there is a major treatment gap for patients whose cancer no longer responds to standard therapies," said Marcia Brose, M.D., Ph.D., principal investigator of the DECISION trial, Assistant Professor in the Abramson Cancer Center and the Perelman School of Medicine at the University of Pennsylvania, and a Medical Advisor of ThyCa: Thyroid Cancer Survivors’ Association.
"The DECISION results demonstrate sorafenib’s ability to extend progression-free survival compared to placebo in these patients, potentially providing a new treatment option for these patients," she added.
The drug’s manufacturer, Bayer HealthCare Pharmaceuticals and Onyx Pharmaceuticals, Inc. reported that these results mean that a supplementary New Drug Application for sorafenib for the treatment of RAI-refractory differentiated thyroid cancer will be submitted in mid-2013 to the U.S. Food and Drug Administration, with additional submissions to follow globally. Sorafenib is currently approved in the U.S. for the treatment of patients with unresectable hepatocellular carcinoma and for the treatment of patients with advanced renal cell carcinoma. It is also approved in 100 countries.
“In the past 30 years, there have been no new treatment options approved for patients with radioactive iodine-refractory differentiated thyroid cancer,” said Pablo J. Cagnoni, M.D., Onyx Pharmaceuticals Executive Vice President, Global Research & Development and Technical Operations, “We are pleased with the results of the DECISION study, which demonstrate sorafenib’s activity in patients who have no other treatment options.”
Thyroid cancer has become the fastest-increasing cancer in the world in recent years and is the sixth most common cancer in women. There are more than 213,000 new cases of thyroid cancer annually worldwide. While the majority of differentiated thyroid cancers are treatable, RAI-refractory locally advanced or metastatic disease is more difficult to treat and is associated with a lower patient survival rate.