I told you so. Analyst Sees Early Negativity At Chiron/Cephalon Hearing
By Anita Womack
BETHESDA, Md. (Dow Jones)--Food and Drug Administration advisory committee hearings usually
unfold slowly, with panel members reluctant to tip their hands until late in the day.
That may not be the case with a new-drug application from Cephalon Inc. (CEPH) and Chiron Corp.
(CHIR) for Myotrophin, a drug to treat amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease.
Dr. Paul Leber, director of the FDA's division of neuropharmacological drug products, began Thursday's
session by defending the agency's goal that a company show substantial evidence that a drug works,
preferably in two confirmatory trials.
''This is not some bureaucratic rule to delay marketing of a product,'' Leber said, adding that in the
current application, ''ordinary standards of evidence have not been met.'' Leber told panel members that
one study failed to confirm the results of the other. The companies are asking us to ''surrender to
modifying our standards,'' Leber said.
Leber said that broadly speaking, efficacy in clinical trials can sometimes be explained by fraud, bias
and chance, as well as by treatment effect. While the agency doesn't think fraud is involved in the
current application, Leber said officials are not sure if bias or chance is involved. It is ultimately up to
the panel to make recommendations to the agency, which is not bound by but usually follows them.
Meanwhile, company officials told members of the FDA's Peripheral and Central Nervous System
Advisory Committee that Myotrophin works differently than other drugs aimed at ALS by targeting the
total motor-neural system.
Chiron and Cephalon currently have a treatment Investigational New Drug application approved in which
patients receive the drug on a compassionate basis and the company makes no money. The company
must now convince the same panel which gave it treatment IND status to approve it for prescription
sales. Many analysts believe that Myotrophin will be cleared primarily because the company has a
treatment IND approved, and the disease itself is so devastating.
But analyst Ira Loss of HSBC Washington Associates said the issues discussed so far Thursday
parallel the discussion in June of last year, adding that the agency seems negative given the lack of a
confirmatory study. ''So far it does not appear that Cephalon has convinced the panel, but it is early.''
Chiron officials told the panel that ALS patients typically have a shorter survival rate than AIDS and
cancer patients. The company added that Myotrophin has a beneficial regenerative effect on nerves,
and can increase muscle mass and strength. Panel members repeatedly asked for proof that
Myotrophin, when injected subcutaneously in the leg of rats and mice, would stimulate sprouting of
nerve cells beyond that point.
An estimated 30,000 Americans suffer from ALS, a disease in which patients slowly lose muscular
function, and often die within five years of diagnosis. The average age of onset of the disease is
mid-50s.
Rhone-Poulenc Rorer (RPR) has the only FDA-approved drug on the market to treat ALS. |
