Cephalon Chief On FDA Panel Decision

In the first face-to-face interview since the US Food and DrugAdministration's advisory panel delivered its second verdict on Cephalon's Myotrophin (mecasermin; Marketletter May 19) for amyotrophic lateral sclerosis, chief executive of the firm Frank Baldino has given the Marketletter his reaction to the announcement.

It is a great disappointment, for Cephalon but especially for the patients, their families and caregivers and the specialists in amyotrophic lateral sclerosis who voiced their support for the drug at the panel meeting, said Dr Baldino. Asked whether he thought the FDA might go ahead and approved the drug, he said: "I'm not sure of anything right now."

The panel was not asked to give a verdict on approval, only on whether there was "substantial" efficacy, he noted, adding that it had a difficult job to do, as ALS is an extremely complex disease and there is still much which is not understood about its pathology. Faced with one significant US study and another European one which showed a trend to efficacy, the panel took the cautious path. Unfortunately, this approach may act as a disincentive to companies trying to develop drugs for hard-to-treat, orphan conditions like ALS, said Dr Baldino.