After a setback four months ago in efforts to move Myotrophin ontothe U.S. market for Lou Gehrig's disease, Cephalon Inc. and itsdevelopment partner, Chiron Corp., said Tuesday they will submit anew drug application (NDA) for the treatment this summer.
Cephalon's stock (NASDAQ:CEPH) jumped $3.62 to close Tuesdayat $31.50, a 13 percent increase. Chiron (NASDAQ:CHIR) ended theday at $96, up $1.25.
In January 1996 FDA concerns about data from one of two Phase IIItrials of Myotrophin, which is recombinant human insulin-like growthfactor, scuttled an attempt by Cephalon to get approval of a treatmentinvestigational new drug (IND) application.
A treatment IND would have made the drug widely available topatients suffering from amyotrophic lateral sclerosis (ALS), or LouGehrig's disease, pending an FDA decision on the NDA, whichCephalon hoped to file in early 1996.
Cephalon officials had said the FDA questioned whether results of asecond Phase III trial in Europe supported findings from the firstPhase III North American study. In both evaluations Myotrophinachieved statistical significance in slowing progression of ALS.However, data from the European trial was not as strong as the NorthAmerican study.
Investor speculation on the depth of the FDA worries sent Cephalon'sstock plummeting 35 percent Jan. 19, 1996, from $35.88 to $23.38.Chiron dipped 5 percent the same day. (See BioWorld Today, Jan.22, 1996, p. 1.)
Cephalon did not know how the FDA concerns would impact theMyotrophin NDA until last week when its representatives met withofficials of the regulatory agency.
Cephalon's Jason Rubin, vice president of corporatecommunications, said Tuesday the FDA told the company it wouldaccept the Myotrophin NDA for review and the drug would bescheduled for an advisory panel hearing. Rubin said the NDA likelywould be filed during the third quarter of 1996.
The FDA did not require a third Phase III trial and safety ofMyotrophin was not an issue, Rubin said.
Some analysts had speculated another pivotal study would haveconstituted a doomsday scenario for Cephalon, delaying the drug'sentry onto the market by several years. Cephalon's share price soaredalmost 400 percent the last half of 1995 based on the two positivePhase III studies and potential approval of Myotrophin in 1996.
Neither Cephalon, of West Chester, Pa., nor Chiron, of Emeryville,Calif., would provide details of the FDA meeting.
In January when the FDA rejected the treatment IND analystsspeculated the agency had not seen all the data from the EuropeanPhase III study.
Tim Wilson, analyst with UBS Securities in New York, said the FDAlast week got a chance to see most of the data that will be included inthe NDA and he suggested Myotrophin will be approved this year.
ALS is a fatal degenerative neuromuscular disease that affects anestimated 70,000 people worldwide. The only FDA approvedtreatment is Rilutek, which is sold by Collegeville, Pa.-based Rhone-Poulenc Rorer and was cleared for marketing in December 1995.
Wilson speculated Myotrophin will outperform Rilutek, whose slowsales have caused some analysts to urge caution on the chances formarket acceptance of Cephalon's drug.
However, Wilson observed, "Saying Myotrophin won't sell becauseRilutek hasn't sold is like saying nobody bought the Edsel so whybuy an automobile."
Wilson predicted Myotrophin revenues for 1997, its first full year onthe market, of $148 million. n
-- Charles Craig
(c) 1997 American Health Consultants. All rights reserved.