Cephalon Inc. announced Thursday that modafinil, itstreatment for narcolepsy, has been designated an orphan drugby the FDA.

Modafinil was approved for marketing in France in February1992. The developer of the drug, Laboratoire L. Lafon of Paris,will begin sales upon pricing and reimbursement approval.

In February, Cephalon (NASDAQ:CEPH) of West Chester, Pa.,licensed from Lafon the rights to develop, market and sellmodafinil in the U.S. and Mexico.

Narcolepsy, a debilitating multigenic illness that affects about125,000 people in the U.S., is characterized most notably byuncontrollable daytime sleep attacks. These attacks can takeplace "in a wink," often occurring while the afflicted individualis in midsentence or in the process of lifting a food-laden forkto the mouth. There's no cure for the disorder, and it isnormally treated by dosing with amphetamines oramphetamine analogs such as Ritilin.

The double-blinded and placebo-controlled European clinicaltrials on modafinil demonstrated that the drug significantlyreduces daytime sleep attacks and drowsiness without adverseside effects.

Now that it has orphan drug status on the drug, Cephalonintends to seek U.S. regulatory approval for modafinil. "We arerapidly advancing this product through the developmentprocess, and expect to file an investigational new drug (IND)application later this year," said Frank Baldino, president andchief executive officer of Cephalon.

He added that Cephalon "can use the French data" to add to thedata it generates in the U.S. trials. "We don't have to do thepreclinical work," he said, but it will be necessary to doaddiction/liability studies, as well as Phase II and III trials.

"It's nice to have a product that's already been shown to work,"Baldino told BioWorld. "This is the first new product in 30years for treating narcolepsy."

It's not entirely clear how modafinil works, but "it's probablyan alpha-1 antagonist," said Christian Guilleminault, a physicianat the Stanford University Sleep Center in Palo Alto, Calif.

Most of the data come from studies on narcoleptic dogs (anatural occurrence) and other laboratory animals. They haveindicated that the brains of narcoleptic individuals have adecreased number of alpha adrenergic receptors.

But "that's not the only defect found. ... The neuronal circuitryis fairly complex," Guilleminault told BioWorld. "Modafinilseems to have some alpha 1-aminergic properties and alsosome dopa-aminergic properties."

-- Jennifer Van Brunt Senior Editor

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