Why not buy RPR stock? They have a produst approved for ALS!! A spokesman for Rhone-Poulenc Rorer, said Rilutek, the only FDA-approved treatment for ALS, had $8 million in sales during the first quarter of 1997. Rilutek was approved in December 1995 and is currently approved in 21 countries. FDA Reviewers Suggest Panel Not Back Myotrophin
BETHESDA, Md. (Dow Jones)--Food and Drug Administration medical reviewers, citing insufficient
evidence on the matter of efficacy, told an advisory panel it shouldn't recommend for approval Chiron
Corp.'s (CHIR) and Cephalon Inc.'s (CEPH) Myotrophin drug for Lou Gehrig's disease.
''Strong evidence in a single study can warrant approval,'' said Dr. John Feeney, medical officer for the
agency's Division of Neuropharmacological Drug Products, told members of the FDA's Peripheral and
Central Nervous System Drugs Advisory Committee. ''But results of (one of two Myotrophin studies)
are not strong. It just barely reaches statistical significance.''
Cephalon Inc. officials presented data on two studies. The first showed significance but was 'not
strong,' agency officials said, while the second did not show significance at all.
Meanwhile, company officials attempted to prove to the FDA panel that there was a regulatory basis to
support approval even if there was not enough data to support an approval.
Bruce Scharschmidt, vice president clinical affairs of Chiron Corp., told the panel that ALS was
designated by the FDA in 1988 as a severely debilitating and life-threatening disease. He added that
regulatory laws permit approval of such drugs if no other treatment is available. Cephalon then
attempted to prove that Myotrophin was different from Rhone-Poulenc Rorer's (RPR) Rilutek because
Myotrophin slows progression of the disease and Rilutek doesn't.
Cephalon officials said Myotrophin slows progression of the disease by 26%.
Myotrophin currently is used as a treatment Investigation New Drug (IND) in which patients can obtain
the drug on a compassionate basis. The company makes no money from treatment IND use.
Cephalon told the panel that 450 patients have been given the drug under the program, but said they
could not collect pertinent data because patients were enrolled on a lottery basis. Patients typically
used in clinical trials are chosen based on established criteria.
Cephalon officials told members of the FDA's Peripheral and Central Nervous System Drugs Advisory
Committee that a nine-month trial reveals that the drug provides a three-month slowing of the loss of
function. Specifically, officials say there was a 20-month median survival with Myotrophin. The survival
rate for patients on placebo was 17 months.
Company officials added that age and worsening pulonary function contributed signficantly to deaths
reported.
Amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease, is a disease in which patients slowly
lose muscular function. Patients typically survive only five years after diagnosis.
Potential 1997 Myotrophin sales are about $30 million. However, sales could grow to $168 million.
Meanwhile, Bob Pearson, a spokesman for Rhone-Poulenc Rorer, said Rilutek, the only FDA-approved
treatment for ALS, had $8 million in sales during the first quarter of 1997. Rilutek was approved in
December 1995 and is currently approved in 21 countries. |
