According to a panel of experts advising the U.S. Food and Drug Administration (FDA), the drug Provigil, although effective in treating attention deficit hyperactivity disorder (ADHD) in children and teenagers, is not safe enough to win approval.
The experts say they are most concerned about the risk of skin rashes that can lead to hospitalization and have called on the drugmaker Cephalon to conduct more clinical trials.
Panel chairman Wayne Goodman, a psychiatrist at the University of Florida, believes more patients need to be studied before approval is granted.
The advisory committee voted 12-1 against recommending Provigil as safe for children with ADHD.
Provigil already has approval to treat sleep disorders, but Cephalon is seeking to market it under the name Sparlon for children and teenagers with ADHD.
The ADHD version of the drug, known generically as modafinil, would be a smaller tablet and offer a wider range of doses.
Conditional approval was granted by the FDA in October 2005 for use in ADHD, but lingering concerns about rashes as well as mania, aggression and other possible psychiatric effects prompted the added advice from an expert panel.
Thomas Laughren the FDA's Director of Psychiatry Products has said more work needs to be done to demonstrate a reason for it to be marketed.
Even if the wider use is approved it is almost certain Sparlon will come with the strongest warning possible, which will be at least a black box warning according to Dr. Robert Temple, head of the FDA's Office of Medical Policy.
The recommendation comes only a day after another FDA panel called for all attention deficit drugs to include new information about psychiatric and heart risks even though the data are still unclear; some panel members are apparently uneasy about supporting Sparlon when other treatments are available and are as effective.
This is yet another setback for Cephalon, which has faced problems with its experimental sleep disorder drug Nuvigil as well as the Vivitrol alcoholism treatment it is developing with Alkermes Inc.
The committee has recommended that Cephalon carry out a 3,000-patient trial to determine what risk the drug may pose for the sometimes fatal skin disease Stevens-Johnson Syndrome.
Drug reactions cause nearly all cases of the disease, which can produce widespread blistering and rashes.
The FDA's drug chief, Dr. Robert Temple, said one out of roughly 900 children involved in earlier studies of the drug developed the disease.
The FDA is not required to follow the recommendations of its advisory committees but usually does.