On April 26, 2006 the FDA approved orphan drug status for sorafenib tosylate (Nexavar tablets. The approval was based in part on data from a phase 2, single-agent study showing that 43% of patients receiving sorafenib experienced stable disease for at least 4 months; an additional 9% of patients experienced tumor shrinkage.
Results from a late-breaking abstract of a phase III trial presented at the 2007 annual meeting of the American Society of Clinical Oncology (ASCO) indicate that Nexavar (sorafenib) significantly improves survival and doubles the time to progression among patients with advanced hepatocellular carcinoma (HCC). This is the first agent to ever demonstrate a significant improvement in survival in the treatment of HCC. An independent data monitoring committee (DMC) that reviewed the clinical data has attested this year to the safety and efficacy of the product and therefore recommended early conclusion of the said study. As a result of this recommendation, Bayer and Onyx stopped the trial and allowed all patients enrolled in this trial access to Nexavar. It is now FDA approved and this recommendation is reflected in the NCCN guideline.
Usha Dutta (2000) Treatment of hepatocellular carcinoma Journal of Gastroenterology and Hepatology 15 (8), 822–824.
A. X. Zhu, D. Sahani, A. Norden-Zfoni, N. S. Holalkere, L. Blaszkowsky, D. P. Ryan, J. W. Clark, K. Taylor, J. V. Heymach, K. Stuart Journal of Clinical Oncology, 2005 ASCO Annual Meeting Proceedings. Vol 23, No. 16S, Part I of II (June 1 Supplement), 2005: 4120, updated as Abstract No: 4637, Journal of Clinical Oncology, 2007 ASCO Annual Meeting Proceedings Part I. Vol 25, No. 18S (June 20 Supplement), 2007: 4637