Bayer/Onyx: Nexavar Approval in Liver Cancer on the Horizon

Bayer and Onyx’s Nexavar has been granted priority review for hepatocellular carcinoma from the FDA and a final decision regarding its approval is now expected in the next few months. The drug has been shown to significantly extend overall survival in previously untreated patients, a population with a high unmet need, ensuring adoption as the standard of care for this patient group.

Following the submission of a supplemental New Drug Application (sNDA) in June 2007, the FDA has now granted Nexavar (sorafenib) priority review for hepatocellular carcinoma (HCC), the most common form of liver cancer. The sNDA was based on data from the Phase III SHARP (Sorafenib HCC Assessment Randomized Protocol) trial in which Nexavar significantly extended overall survival by 44% in previously untreated HCC patients versus those taking placebo.

Median overall survival was 10.7 months in Nexavar-treated patients compared to 7.9 months for those taking placebo in this pivotal trial which involved 602 advanced HCC patients. As a result, Bayer and its US partner Onyx Pharmaceuticals halted the trial in February 2007 and allowed all enrolled patients to continue receiving Nexavar treatment.

Priority review status is intended to expedite the regulatory review process for agents that address unmet medical needs. Based on this designation, the FDA reviews the application with a goal of taking action within six months from the date on which they received the sNDA. If approved, Nexavar would be the first FDA-approved therapy for HCC.

HCC is the most common form of liver cancer and is responsible for about 90% of the primary malignant liver tumors in adults. It is the fifth most common cancer in the world and the third leading cause of cancer-related deaths globally. The disease is associated with hepatitis infection, and so is most common in developing countries where hepatitis B and C infections are endemic. Despite this high prevalence, treatment options are limited and no effective systemic therapies exist. Indeed, there also appears to be a lack of consensus regarding prognosis and therapeutic options. The ability of Nexavar to confer a statistically significant overall survival benefit in the first-line treatment setting for metastatic HCC patients is therefore a major breakthrough.

Given the high unmet need in HCC, it is likely that Nexavar will be granted approval and this will be well received with encouraging uptake. Nexavar’s sales are expected to be significantly boosted, since the drug is currently only indicated for treatment of metastatic renal cell carcinoma. However, with an expensive price-tag, Bayer and Onyx will need to effectively communicate the value of Nexavar if it is to penetrate the more cost-conservative pharmaceutical markets to any real extent.