FDA committee gives thumbs down to drug Genasense By Karl Stark Knight Ridder Newspapers PHILADELPHIA — An advisory committee for the U.S. Food and Drug Administration Monday (May 3, 2004) declined to support approving the anti-cancer drug, Genasense, developed more than a decade ago at the University of Pennsylvania. The advisory committee recommendation, which is usually followed by the agency, dealt a blow to the drug’s prospects and to Genta Inc. of Berkeley Heights, N.J., and Aventis SA, the compound’s developer and marketer. Aventis had predicted that Genasense would sell more than $2 billion a year, and Penn stood to reap tens of millions of dollars for licensing the underlying technology. But those hopes appear dashed — at least for now. Genta is testing the drug in 20 studies, including one focusing on melanoma, or skin cancer, which the advisory committee ruled on Monday. “We’ll continue the clinical development of Genasense,” Genta spokeswoman Joy Schmitt said. Trials involving two blood-borne cancers, chronic lymphocytic leukemia and multiple myeloma, are in phase III, which are pivotal tests for getting new drugs approved. But Quynh Pham, a biotech analyst for Delafield Hambrecht in Seattle, said Genta executives had promised to go to the FDA with the strongest results from one of its three Phase III clinical trials. “They chose melanoma with no explanation so it makes you a little skeptical,” she said. “We’re cautious. We think the company has a high chance of Genasense not getting approved.” Louis P. Berneman, managing director for Penn’s Center for Technology Transfer, predicted it would take six to 12 months for the companies to address the FDA’s questions. “There is much whitewater in the path from discovery to product launch,” Berneman said. “This represents some more whitewater. We are hopeful that Genta and Aventis will steer a clear course, and get a different response as early as the end of the year.” Genasense, known also as oblimersen sodium, was discovered by John C. Reed while he was an associate professor at Penn from 1989 to 1992. Reed is currently chief executive of the Burnham Institute, a research center in San Diego. Genasense works by blocking a protein that keeps cells from dying. The drug, which is administered via chemotherapy, makes cancer cells more vulnerable to death. Several doctors not connected with the company said last week that Genasense appeared relatively nontoxic and could help in a broad range of cancers. The FDA granted Genasense a priority review in February and promised to make a final decision before June 8. On Monday 11 of the 16 members of the FDA oncology panel agreed that the small but observed differences in response were real. But the vote was more negative on whether the drug represented a gain in survival without the disease worsening. Only four voted “yes” while 12 voted “no.” And only three of 13 said the drug’s benefits outweighed its added toxicity.