"FDA Grants Orphan Drug Designation to Nexavar (Sorafenib) for the Treatment of Hepatocellular Carcinoma"
WEST HAVEN, C.T., and EMERYVILLE, C.A. -- April 26, 2006 -- Bayer Pharmaceuticals Corporation and Onyx Pharmaceuticals, Inc. announced today that Nexavar(R) (sorafenib) tablets has been granted orphan drug status for the treatment of hepatocellular carcinoma (HCC), or liver cancer, by the U.S. Food and Drug Administration (FDA). A similar designation has been granted by the European Commission.
"We are very encouraged by the collaboration of the FDA as well as the European Regulatory bodies in our research surrounding this highly fatal disease," said Susan Kelley, M.D., vice president, Oncology, Bayer Pharmaceuticals Corporation. "It fuels our efforts to strive toward a novel therapy for liver cancer patients worldwide."
At the 16[th American Association for Cancer Research-National Cancer Institute-European Organization for Research and Treatment of Cancer (AACR-NCI-EORTC) meeting in 2004, investigators reported that in a phase 2 single agent study, 43% of patients treated with Nexavar experienced stable disease for at least four months and an additional nine% of patients experienced tumor shrinkage.
The most common grade 3/4 drug-related toxicities were fatigue (9.5%), diarrhea (8%), and hand-foot skin reaction (5%). The toxicity profile of Nexavar was similar to previously reported safety analysis in patients with renal cell carcinoma.
A Phase III trial of Nexavar administered as a single agent is ongoing. It is designed to measure differences in overall survival, time-to-symptom progression and time-to-tumor progression of Nexavar versus placebo in liver cancer patients. A randomized phase 2 trial for liver cancer patients to evaluate the efficacy of Nexavar in combination with the chemotherapeutic agent doxorubicin is also ongoing.
"Liver cancer is such an aggressive disease that patients diagnosed with it rarely live beyond two years," said Dr. Jordi Bruix, head of the Barcelona Clinic Liver Cancer Group at the University of Barcelona in Spain. "The global medical community recognizes the need for new treatments in liver cancer and I am hopeful of the potential of Nexavar in this patient population." Dr. Bruix is co-primary investigator of the Phase III trial along with Dr. Josep Llovet, senior scientist of the division of liver disease at Mount Sinai School of Medicine in New York.
In December 2005, Nexavar received approval from the FDA to treat patients with advanced renal cell carcinoma (RCC), or kidney cancer. The European Commission has also granted an orphan medicinal product designation for Nexavar in renal cell carcinoma and a Marketing Authorization Application (MAA) had been submitted to the European Medicines Agency (EMEA) in September 2005 for treatment of patients with kidney cancer with Nexavar within the European Union.
Orphan Drug Designation in the United States
In the United States, the Orphan Drugs Act (ODA) provides for the orphan drug designation which aims to encourage the development of drugs involved in the diagnosis, prevention or treatment of a medical condition affecting fewer than 200,000 people in the country. The designation grants U.S. market exclusivity to a drug for a particular indication for a seven-year period if the sponsor complies with certain FDA specifications. Additional incentives for the sponsor include tax credits related to clinical trial expenses and a possible exemption from the FDA-user fee. The designation does not shorten the duration of the regulatory review and approval process.
About Nexavar
Nexavar is the first oral multi-kinase inhibitor that targets both the tumor cell and tumor vasculature. In preclinical models, Nexavar targeted members of two classes of kinases known to be involved in both tumor cell proliferation (tumor growth) and tumor angiogenesis (tumor blood supply) -- two important cancer growth activities. These kinases included RAF kinase, VEGFR-2, VEGFR-3, PDGFR-B, KIT, and FLT-3.
Nexavar is currently in Phase III clinical trials for the treatment of liver cancer, metastatic melanoma, or skin cancer, and non-small cell lung cancer (NSCLC), and has been studied in more than 20 tumor types and in more than 8,000 clinical trial patients. In addition to company-sponsored trials, there are a variety of Nexavar studies being sponsored by government agencies, cooperative groups and individual investigators.
About Hepatocellular Carcinoma
Hepatocellular carcinoma (HCC), also known as primary liver cancer, is the most common form of liver cancer and is responsible for 80% of the primary malignant liver tumors in adults. It is the fifth most common cancer in the world. HCC disproportionately affects men, with four times as many men developing the disease as women. In 2002, approximately 626,000 cases of HCC were reported worldwide (15,000 in the United States and 53,600 in Europe), and more than 600,000 deaths (about 13,000 Americans and 57,000 Europeans) due to HCC were reported. The five-year relative survival rate is about 7%.
Important Safety Considerations for U.S. Patients Taking Nexavar
Based on the current, approved package insert for the treatment of patients with advanced kidney cancer, hypertension may occur early in the course of therapy and blood pressure should be monitored weekly during the first six weeks of therapy and treated as needed. Incidence of bleeding regardless of causality was 15% for Nexavar vs. 8% for placebo and the incidence of treatment-emergent cardiac ischemia/infarction was 2.9% for Nexavar vs. 0.4% for placebo.
Most common treatment-emergent adverse events with Nexavar were diarrhea, rash/desquamation, fatigue, hand-foot skin reaction, alopecia, and nausea. Grade 3/4 adverse events were 38% for Nexavar vs. 28% for placebo. Women of child-bearing potential should be advised to avoid becoming pregnant and advised against breast-feeding. In cases of any severe or persistent side effects, temporary treatment interruption, dose modification or permanent discontinuation should be considered.
Nexavar(R) (sorafenib) tablets is a registered trademark of Bayer Pharmaceuticals Corporation.
SOURCE: Bayer Pharmaceuticals Corporation; Onyx Pharmaceuticals
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