Wall Street Journal Front page: Clinical Trial Does Not Prove Myotrophin's Value, Panel Says
By ANITA WOMACK and ELYSE TANOUYE
Staff Reporters of THE WALL STREET JOURNAL
A Food and Drug Administration panel voted 6-3 to reject
Chiron Corp.'s and Cephalon Inc.'s new-drug application for
Myotrophin for the treatment of Lou Gehrig's disease.
Members of the FDA's Peripheral and Central Nervous
System Drug Advisory Committee said the application had
only one adequate and well-controlled clinical trial to prove
the drug's efficacy. "The data don't look robust," said the
panel's chairman, Dr. Sid Gilman of the University of
Michigan Medical Center.
The FDA isn't bound by the panel's decision, but generally
follows its lead. In this case, the agency has long held
concerns about the Myotrophin data and probably will ask
for more convincing proof that the drug actually works.
It isn't clear that the companies are willing to continue to
pour money into the drug. Cephalon officials told the panel
that more than $130 million had been spent studying the
disease, formally known as amyotrophic lateral sclerosis.
They said the company was willing to do additional
studies, provided they first received the approval.
Stock Reflects Uncertainty
The outcome of the meeting had been uncertain, and
Cephalon's stock had reflected it, although its share price
had risen in recent days on growing optimism that meeting
would go well. Cephalon, based in West Chester, Pa., has
other drug prospects in its pipeline, but Myotrophin was
the company's lead compound and is clearly important to
its financial future; Chiron, a much bigger company with
headquarters in Emeryville, Calif., will suffer less from the
setback.
Responding to the committee's vote, Frank Baldino Jr.,
Cephalon's president and chief executive officer, and
William Rutter, Chiron's chairman, said in a joint
statement: "We are deeply disappointed with the
committee's finding about Myotrophin. We appreciate the
many expressions of support from patients, caregivers and
physicians and will be reviewing with the FDA the panel's
recommendation."
The Myotrophin decision may also affect the biotechnology
sector in general, as Cephalon has been viewed as a
bellwether of the industry. Its announcement in 1995 of
positive results in its first Myotrophin clinical trial lifted the
spirits -- and stock prices -- of the entire biotechnology
industry, which had been battered by a series of drug
failures.
Concern Over Second Trial
Myotrophin's troubles began late last year when the FDA
became concerned about the companies' second trial,
conducted in Europe. The companies said in the fall that
the trial's results were positive, but the agency refused to
approve the their application to get the drug to patients
before full FDA approval -- a status called "treatment IND."
Instead, the agency referred the matter to an expert panel.
At the panel meeting last June, the FDA and some panel
members disputed the companies' methodologies and
conclusions. That panel recommended approval of the
treatment IND, but some members strongly suggested that
Cephalon and Chiron conduct a third trial before seeking
full marketing approval. But Cephalon said the companies
couldn't justify another study economically before receiving
full FDA approval.
At Thursday's meeting, FDA officials again raised
questions about the data.
"We are as anxious as nearly everyone afflicted [by the
disease] in the audience," said Dr. David A. Drachman,
professor of neurology at the University of Massachusetts
Medical School. "My sense is we have seen cosmetic
changes in the data, nothing substantial. We find
ourselves where we were last June -- we want more data. I
am not sure how I am supposed to change my judgment. I
feel squeezed."
Thursday's vote is the latest in a series of setbacks in the
search for drugs to treat the debilitating disease, which
causes progressive paralysis and eventually death. About
30,000 people are estimated to be afflicted with the
disease in the U.S. In recent years, several biotechnology
companies have suffered high-profile failures in their
attempts to use nerve-growth factors such as Myotrophin
to treat the disease. But a different type of drug,
Rhone-Poulenc Rorer Inc.'s Rilutek, won FDA approval in
1995 to treat the malady.
A U.S. Food and Drug Administration
advisory panel voted 6-3 to reject Chiron's
and Cephalon's new-drug application for
Myotrophin to treat Lou Gehrig's disease. |
