Bayer Pharmaceuticals and Onyx Pharmaceuticals' investigational agent—sorafenib (BAY 43-9006)—demonstrated efficacy and was well tolerated in an ongoing phase III trial in patients with advanced renal carcinoma. The study results were presented in an oral session during the 41st annual meeting of the American Society of Clinical Oncology (ASCO) held last month in Orlando, Fla. Sorafenib is the first oral multi-kinase inhibitor that targets serine/threonine and receptor tyrosine kinases in both the tumor cell and tumor vasculature. In preclinical trials, sorafenib has demonstrated both antiproliferative and anti-angiogenic properties.
Bayer Pharmaceuticals and Onyx Pharmaceuticals' investigational agent-sorafenib (BAY 43-9006)-demonstrated efficacy and was well tolerated in an ongoing phase III trial in patients with advanced renal carcinoma. The study results were presented in an oral session during the 41st annual meeting of the American Society of Clinical Oncology (ASCO) held last month in Orlando, Fla. Sorafenib is the first oral multi-kinase inhibitor that targets serine/threonine and receptor tyrosine kinases in both the tumor cell and tumor vasculature. In preclinical trials, sorafenib has demonstrated both antiproliferative and anti-angiogenic properties.
Over 900 patients with advanced renal carcinoma, who had previously failed one prior systemic therapy, were randomized to receive either 400 mg sora-fenib or placebo twice daily. Progression-free survival was doubled to a median value of 24 weeks in patients receiving sorafenib compared with 12 weeks for patients receiving placebo. According to the firms, this is the first randomized study demonstrating that an oral targeted agent significantly delays disease progression in patients with advanced kidney cancer.
The companies are currently preparing a New Drug Application (NDA) for possible approval by the Food & Drug Administration, and they expect to receive accelerated approval by the first half of 2006 based on the results from this phase III study.
Constipation is one of the most common disorders that is suffered by Americans, and it affects between 2% and 27% of the population. Chronic constipation is a disorder of colon motility, which is present for at least three months and results in infrequent bowel movements and in demonstrated difficulty in evacuating stools.
Lubiprostone, which is administered orally, works locally in the gastrointestinal tract by activating specific chloride channels on cells lining the small intes-tine, thereby increasing intestinal fluid secretion.
According to the firm, in two phase III trials, lubiprostone produced rapid and sustained effects in treating the symptoms of constipation. In addition, the results from a randomized withdrawal study indicated no rebound effect after treatment discontinuation.
Additional trials are ongoing. They are investigating the use of lubiprostone for constipation-predominant irritable bowel syndrome and other GI disorders. If approved, Takeda Pharmaceuticals' U.S. subsidiary will market lubiprostone in the United States.
Fast track awarded to first once-daily oral iron chelator Severely anemic patients suffering iron overload from frequent blood transfusions may soon be able to treat the condition with a daily pill rather than lengthy transfusions with deferoxamine mesylate, the current standard of care for iron chelation.
Novartis has filed a new drug application for deferasirox (Exjade), also referred to as ICL670. The agent is a once-daily oral iron chelator for the treatment of chronic iron overload due to blood transfusions. If left untreated or undiagnosed, iron overload can ultimately damage the liver, heart, and endocrine glands.
While treatment with deferoxamine mesylate is effective, it usually requires subcutaneous infusion lasting eight to twelve hours a day, for five to seven days a week for as long as the patient continues to receive transfusions.
The filing is based on results from worldwide clinical trials involving more than 1,000 patients. Novartis reported that in a phase III head-to-head trial comparing deferasirox and deferoxamine mesylate, deferasirox significantly reduced liver iron concentration in both adult and pediatric patients who were receiving blood transfusions.
Therapy with deferasirox was found to be generally well tolerated, with the most frequently reported adverse events being nausea, vomiting, diarrhea, abdominal pain, skin rash, and mild stable increases in serum creatinine, usually within the normal range.
The FDA had previously granted the novel therapy fast-track and orphan drug status. The agency must also give its approval to the proposed brand name, Exjade. Analysts expect the drug to be launched in 2006.