By Lisa Seachrist
Cephalon Inc. said Tuesday that it has submitted its lead narcolepsy drug, Provigil (modafinil), to the FDA for marketing clearance. This is the first new drug application (NDA) the West Chester, Pa., company has filed with the agency.
"This is an exciting new type of drug," said Frank Baldino Jr., Cephalon's president and CEO. "Coupled with the pending NDA of Myotrophin, this is a pretty exciting time for us."
Modafinil is a novel chemical that is approved in France where it was developed to treat narcolepsy and idiopathic hypersomnia by Laboratoire L. Lafon, a pharmaceutical company based in Paris. Cephalon has exclusive rights to market the drug in the U.S., Japan, Ireland, the U.K. and Mexico. In 1993 the FDA granted the company orphan drug status for the treatment of narcolepsy.
Narcolepsy affects approximately 125,000 people in the U.S. A chronic, lifelong, sleep disorder, narcolepsy causes people to constantly flux between sleeping and waking. They not only have a hard time staying awake, but also find that they can't stay asleep. As a result, the most common symptom is excessive sleepiness during the day.
"Narcolepsy is often considered a joke, but people who suffer from the disorder have such difficulty staying awake that they have a hard time maintaining a job or their social relationships," said sleep researcher Joyce Walsleben, director of the New York University Sleep Disorder Center. "If they are in school, they are often mislabeled as lazy or retarded and at work they often fall asleep so often that they aren't productive."
Currently, narcoleptics are treated with amphetamines or amphetamine-like drugs. However, those drugs produce dramatic ups and downs as the medication enters and exits the bloodstream. Some people experience cardiac side effects that prevent them from taking the drugs. In addition, some patients have become addicted to the amphetamines.
While Provigil doesn't show any amphetamine characteristics or side effects, its exact mode of action is unknown. "We certainly know how it doesn't work, but we haven't pinpointed its mechanism of action," said Kori Beer, manager of corporate communications with Cephalon.
The company submitted the results of two Phase III clinical trials in 558 patients with narcolepsy as part of the NDA. The first study was a double-blind placebo controlled study of 285 patients at 18 U.S. medical centers. Patients either received 200 mg or 400 mg of Provigil or a placebo for nine weeks. The patients on the highest dose stayed awake 76 percent longer than those who received placebo. Patients getting the lower dose stayed awake 61 percent longer.
"The side effects of this drug were so minor that almost all of the patients thought they were on placebo at first," said Walsleben who participated in the trial. "That is until they started to receive compliments about their performance at work or their spouses started to notice that they were more alert."
The second trial also tested the two doses of Provigil against placebo for nine weeks followed by a two week observation period in 273 patients. Those on Provigil stayed awake approximately 50 percent longer than those on placebo.
"This drug is really exciting because it is the first new drug to treat narcolepsy in almost 20 years," Walsleben said. "And, these people are desperate for help."
Cephalon's NDA filing is exciting for investors as well, said Peter Ginsberg, an analyst with Vector Securities International. "This is a very important announcement because they are filing Provigil before the year's end," he said.
Ginsberg noted that "Provigil has a clear advantage over current therapies in both safety and efficacy."
Ginsberg expects that Provigil will gain approval by early 1998 and will garner at least $55 million for the company in 2000.
"I like the stock quite a lot and Cephalon is clearly the leading biotech focused in neuroscience," Ginsberg said. "Cephalon has filed Provigil and will file Myotrophin * their ALS drug * in the first quarter of 1997. There aren't many companies with two major drugs filed with FDA at the same time."
Cephalon's stock (NASDAQ:CEPH) was $20.25, up $1.75 on the news. *