* FDA advisory panel votes 10-4 against approval of Xolair
* Experts concerned over safety; split on efficacy
* Move means Xolair may stay restricted to over-12s (Adds comment from companies)
By Ben Hirschler
SILVER SPRING, Md., Nov 18 (Reuters) - Roche ROG.VX and Novartis’s NOVN.VX asthma drug Xolair should not be approved for children aged 6 to 11, a U.S. advisory panel decided on Wednesday.
Although the drug has shown modest benefits in treating children with particularly bad asthma in clinical tests, experts were concerned about the drug’s safety and the possibility of a long-term cancer link.
Xolair has been cleared since 2003 for use by adults and adolescents aged 12 and older. Roche and Novartis are eager to also offer it in the important U.S. children’s market — but the panel’s decision means this is now unlikely.
The independent committee of Food and Drug Administration advisers voted 10 to 4 against recommending approval of the injectable biotech drug, which is designed for people with moderate to severe asthma as an add-on to standard therapy.
Novartis and Roche unit Genentech said in statement the companies were “disappointed by today’s vote but remain confident” in the safety and effectiveness data submitted to the FDA. An agency decision on the proposed pediatric use is expected in January, the companies said.
Xolair was approved for use in children aged 6 to 11 years in Europe in August.
In 2008, the drug generated sales of $517 million for Genentech and $211 million for Novartis. Novartis and Genentech jointly market Xolair in the United States, while Novartis markets it elsewhere.
The FDA will consider the panel’s input as it decides whether to approve the expanded use of Xolair. The agency usually follows panel recommendations.
Panel chairman Dr. Dennis Ownby, a professor of pediatrics at the Medical College of Georgia, said Xolair would probably help some children, but not enough was known about the safety implications of giving it for prolonged periods to youngsters.
“While this drug may be very useful in some patients, I’m still concerned that we haven’t done adequate diligence on exploring all the possible adverse effects,” he said.
The committee was split evenly on the effectiveness of the drug but voted by 9 to 5 that its safety record had not been adequately assessed.
Thomas Platts-Mills of the University of Virginia Medical Center, one of those speaking up for the treatment, said he believed Xolair’s benefits were real but were hard to prove.
Dr. Peter Starke of the FDA’s pulmonary and allergy products division told the meeting that while the drug had met its targets in clinical trials, its effectiveness in children was “invariably small and clinically modest.”
The drug’s current label for adult use contains a warning that it may cause anaphylaxis — a severe allergic reaction that can be fatal — and could also put patients at risk of cancer.
FDA staff said pediatric studies had not revealed any new safety issues, but there was no reason to think children would not face similar side-effect risks.
Xolair works by disabling a naturally occurring antibody called IgE, which triggers the release of chemicals that cause inflammation and provoke asthma and allergy attacks. The drug’s generic name is omalizumab. (Editing by Bernard Orr and Steve Orlofsky)