By Lisa Seachrist

Washington Editor

WASHINGTON — Cephalon Inc. and Chiron Corp. have withdrawn and resubmitted the new drug application (NDA) for their Lou Gehrig's disease drug, Myotrophin, to the FDA at the request of the agency.

With the Nov. 11 statutory deadline for a decision on the drug approaching, the FDA decided it needed more time to adequately review Myotrophin data even though the agency had postponed the decision by three months in August. As a result, the FDA suggested to the companies that they resubmit the NDA which would give the agency six months to review the data.

"It appears that FDA is making a concerted effort to reach a reasonable conclusion on Myotrophin," said Jason Rubin, vice president of corporate communication for the West Chester, Pa.-based Cephalon. "This resubmission resets the clock for the agency to give them more time to analyze the Myotrophin data."

Rubin noted that the companies have not added any data to the resubmission, and that the resubmission did not involve paying another set user fees. Cephalon and the Emeryville, Calif.-based Chiron are co-developing Myotrophin for the North American and European markets.

Amyotrophic lateral sclerosis (ALS) or Lou Gehrig's disease, is a progressive, fatal, neurodegenerative disease which destroys motor neurons—the nerve cells that control muscle activity. ALS affects about 70,000 people worldwide and leads to progressive loss of muscle control which eventually leads to paralysis respiratory failure and death.

Myotrophin (recombinant human insulin-like growth factor-1) was first submitted to the agency for review Feb. 11 and was given expedited review status under the Prescription Drug and User Fee Act (PDUFA). With this status, the agency had six months to review the drug and render a decision on approval.

In May, an FDA advisory panel refused to recommend Myotrophin for approval citing conflicting clinical results. A North American trial had shown that the drug slowed the progression of ALS by 26 percent; however, a European study of the drug failed to show that it had any benefit at all.

At that May 8 meeting, ALS patients and their families made impassioned pleas to the committee to recommend the approval of the drug. Nevertheless, the panel voted 6-3 against recommending Myotrophin for approval.

In August, with the statutory deadline nearing, with patient advocates protesting at the FDA's Rockville, Md., campus, and with U.S. Sen. Orrin Hatch (R-Utah) urging the agency "to provide ALS patients a drug they need which is at no risk to the public health," the FDA requested additional information from the companies, extending review of Myotrophin by three months.

Having already used their option for extending review under PDUFA, the only way that the agency could continue to consider Myotrophin was to have the companies withdraw and resubmit their NDA.

FDA May Be Split Over Approval

"Clearly there are two factions at FDA, one which is inclined to approve Myotrophin and one that is against it," Tim Wilson, an analyst with UBS Securities, in New York, told BioWorld Today. "Up until today, our view was that Myotrophin had a 25 percent chance of being approved. Now, we think this move raises the chances to 40 percent."

Because most analysts saw the chances for Myotrophin approval to be tremendously low, Wilson noted that the market currently values Cephalon's stock without looking for Myotrophin approval. However, he noted that he wouldn't be surprised if over the next few days Cephalon's stock price goes up.

"This move is indicative of an intensive review at the agency," Wilson said. "And, some of the retrospective data as well as the open label study indicate that this is an active product. It's not the best data, but it is a desperate disease. On balance, I would say that Myotrophin should be approved."

Wilson noted that while the resubmission was unusual, it was the only way that the FDA could legally get more time to review the drug.

Rubin noted that the companies have filed for approval in Europe, and that submission is proceeding on schedule. No European decision is expected until next year.

Cephalon's stock (NASDAQ: CEPH) closed Tuesday at $10.437, down $0.813. Chiron (NASDAQ:CHIR) ended the day at $19.125, down $0.187.

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