Th US FDA has approved the supplemental NDA for the oral multi-kinase inhibitor Nexavar (sorafenib) tablets for the treatment of patients with locally recurrent or metastatic, progressive, differentiated thyroid carcinoma (DTC) refractory to radioactive iodine treatment. Nexavar was approved following a priority review by the FDA, a designation reserved for drugs that may offer a significant improvement in treatment over existing options.

"Locally recurrent or metastatic, progressive, differentiated thyroid carcinoma that is refractory to radioactive iodine treatment is difficult to treat," said Pamela A Cyrus, MD, vice president and head of US Medical Affairs, Bayer HealthCare Pharmaceuticals. "Nexavar is the first and only FDA-approved therapy for this type of thyroid cancer and is a positive development for patients who previously had limited treatment options."

"We are pleased to be able to offer Nexavar as a treatment option for patients with thyroid cancer who are no longer responding to standard therapy," said Pablo J Cagnoni, MD, president, Onyx Pharmaceuticals, Inc. "We are committed to making this treatment available to physicians and their patients as quickly as possible."

"An unmet medical need exists for this type of thyroid cancer, underscoring the need for new therapies," said Gary Bloom, executive director of ThyCa: Thyroid Cancer Survivors' Association, Inc. "We are excited that an FDA-approved treatment is now available to patients coping with this challenging type of thyroid cancer."

The FDA approval is based on the results of the DECISION (stuDy of sorafEnib in loCally advanced or metastatIc patientS with radioactive Iodine refractory thyrOid caNcer) trial, an international, multicenter, placebo-controlled study.

"The DECISION trial results show sorafenib's ability to extend progression-free survival compared to placebo in patients with this type of advanced thyroid cancer," said Marcia Brose, assistant professor in the Department of Otorhinolarlyngology: Head and Neck Surgery and the division of Hematology/Oncology in the Abramson Cancer Center, Perelman School of Medicine, University of Pennsylvania. "Physicians now have an approved treatment option that may help improve care in this patient population."

A total of 417 patients with locally recurrent or metastatic, progressive differentiated thyroid carcinoma refractory to radioactive iodine  treatment were randomized to receive 400 mg of oral sorafenib twice daily (207 patients) or matching placebo (210 patients). Metastases were present in 96 per cent of the patients: lungs in 86 per cent, lymph nodes in 51 per cent, and bone in 27 per cent.

Sorafenib significantly extended progression-free survival (PFS), the primary endpoint of the study. The median PFS was 10.8 months (95% CI 9.1-12.9) among patients treated with sorafenib compared to 5.8 months (95% CI 5.3-7.8) among patients receiving placebo (HR=0.59 [95% CI, 0.46, 0.76]; p<0.001). PFS was evaluated by an independent radiological review committee using modified Response Evaluation Criteria in Solid Tumours (mRECIST).

Nexavar is approved in the US for the treatment of patients with unresectable hepatocellular carcinoma, patients with advanced renal cell carcinoma and patients with locally recurrent or metastatic, progressive, differentiated thyroid carcinoma refractory to radioactive iodine treatment. Nexavar is thought to inhibit both the tumour cell and tumour vasculature. In in vitro studies, Nexavar has been shown to inhibit multiple kinases thought to be involved in both cell proliferation (growth) and angiogenesis (blood supply) - two important processes that enable cancer growth. These kinases include Raf kinase, VEGFR-1, VEGFR-2, VEGFR-3, PDGFR-B, KIT, FLT-3 and RET.

Nexavar is currently approved in more than 100 countries. Nexavar is also being evaluated by Bayer and Onyx, international study groups, government agencies and individual investigators in a range of cancers.

Nexavar is co-developed by Onyx and Bayer, except in Japan where Bayer manages all development. The companies co-promote Nexavar in the U.S. Outside of the US Bayer has exclusive marketing rights, and Bayer and Onyx share profits globally, excluding Japan.

Onyx and Bayer offer a patient assistance program REACH (Resources for Expert Assistance and Care Helpline) for patients who are not able to pay for Nexavar. The companies also provide financial support to co-pay foundations.

Bayer HealthCare Pharmaceuticals Inc. is the US-based pharmaceuticals business of Bayer HealthCare LLC, a subsidiary of Bayer AG. Bayer HealthCare is one of the world's leading, innovative companies in the healthcare and medical products industry, and combines the activities of the animal health, consumer care, medical care, and pharmaceuticals divisions.

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