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Monday August 11 9:51 AM EDT
Cepahlon girds for FDA Myotrophin verdict
By Ransdell Pierson
NEW YORK, Aug 11 (Reuter) - Cephalon Inc is bracing for a decision by the U.S. Food and
Drug Administration, possibly today, on whether to approve Myotrophin, an experimental drug
to slow the progress of Lou Gehrig's disease.
``It's an uphill battle for Cephalon because arguments for Myotrophin from a scientific
point of view aren't very strong,'' said Mehta and Isaly drug analyst Michael Sheffery.
``Based upon data I've seen, it's not clear the drug does much of anything, but most people
agree it does no harm.''
Cephalon and partner Chiron Corp (CHIR) suffered a setback May 8 when an FDA advisory panel
voted 6-3 against recommending approval of the drug for the fatal muscle-wasting disease
formally known as amyotrophic lateral sclerosis (ALS).
Although the FDA usually follows the advice of its advisory panels, Cephalon decided to
press ahead with the New Drug Application it submitted on February 11.
The FDA agreed to review the application on an expedited basis, meaning it would render its
decision within six months rather than after the usual 12-month review period.
``Monday is the deadline so we are hoping to hear the FDA's decision early in the week.
We're hopeful the drug will be approved,'' said Jason Rubin, a spokesman for Cephalon, a
biotech startup based in West Chester, Pa.
``We'd like to begin marketing it as soon as possible after product labeling and
packaging,'' Rubin told Reuters.
Emeryville, Calif.-based Chiron would manufacture the nerve growth drug and both companies
would share equally in profits, he said.
Rubin said about 30,000 people in the U.S. suffer from ALS, which almost always leads to
death within five years as muscles fail to the point that breathing becomes impossible.
In June 1996, the same FDA advisory panel allowed Cephalon limited compassionate-use
distribution of Myotrophin based on statistically significant results of a Phase III North
American study of 266 patients completed in 1995.
Several panel members expressed concern, however, about the drug's failure to achieve
statistical significance in a second trial among 183 patients in Europe. They urged
Cephalon to conduct a third study to clarify the ambiguous data.
But Cephalon refused, giving the panel a ``hardball take it or leave it'' response, said
Sheffery.
``Cephalon essentially said they were not going to put any more money into a third trial
and came back with a new drug application'' in February, the analyst added.
``They came back and filed an NDA based on the same data the advisory panel had already
seen and had expressed their displeasure with,'' he said.
Cephalon's tough approach surprised many Wall Street observers, Sheffery said.
``If you had been at the June 1996 advisory panel meeting, you'd have shaken your head and
said if those guys (Cephalon) don't do a third trial, they're in big trouble,'' he said.
Analysts said Cephalon and Chiron have been flooding the FDA in recent months with
supplementary data from the earlier trials, hoping to bolster their application.
Cowen & Co drug analyst Joyce Lonergan said the FDA was in a ``scary'' position, caught
between its own panel's negative stance and the outspoken desire by many Lou Gehrig's
sufferers and their families for approval of Myotrophin.
Another factor, she said, is pressure for approval by Republican Sen Orin Hatch of Utah,
who urged the FDA in a letter ``to provide patients with a drug they need at no risk to
public health.''
``Nobody makes any friends by taking a stance on this,'' Lonergan said. She added that the
FDA was having to resolve the high-profile drama although no successor has yet been named
to former FDA chief David Kessler, who resigned in March.
Cephalon, whose shares peaked in late 1995 at 40-3/4 on favorable results of its U.S.
pivotal trial, closed Friday at 11-3/16.
Lonergan said shares could rise to between $15 and $20 by late 1998 regardless of
Myotrophin's fate, based on Cephalon's ``extensive pipeline'' of other drugs -- including
narcolepsy drug Provigil, now awaiting FDA approval.
She said approval of Myotrophin would be icing on the cake. Sales of the drug have a
potential annual worldwide market of $500 million, she said.
More news for referenced ticker symbols: CEPH, CHIR, and related categories and
industries: healthcare, stock capsules. |
