Updated from Oct. 2
Shares of
Transkaryotic Therapies plunged 62% Thursday after the company said late Wednesday that efforts to seek U.S. approval for its experimental
Fabry disease drug, Replagal, will be delayed into next year because of
concerns raised by the Food and Drug Administration.
The Cambridge, Mass.-based firm also warned that third-quarter sales
for Replagal in Europe were lower than expected in the current quarter. The
shortfall is forcing TKT to cut its 2002 sales estimate for the drug.
The surprise disclosure by TKT of serious problems with Replagal comes
less than two weeks after the FDA abruptly postponed a Sept. 27 advisory
panel meeting that was scheduled to review the drug. A similar panel for
Genzyme and its Fabry disease drug, Fabrazyme, also was postponed. At the time, the FDA said the panels were postponed because of conflict of interest issues with some of the doctors invited to participate at the panel meeting.
Shares of TKT sank $20.72, or 62%, to $12.53 in recent trading Thursday.
On a conference call to discuss the Replagal setback, TKT CEO Richard Selden came under attack from several angry fund managers who questioned his credibility in the face of prior statements that all was well with the company's FDA discussions. One particularly irate fund manager ended the question-and-answer period of the call by asking Selden if, and when, he expects to be contacted by investigators from the
Securities and Exchange Commission.
TKT finds itself in a particularly vulnerable position because of its competitive battle with Genzyme. The FDA is supposed to render a decision under rules defined by the Orphan Drug Act, which means that only one drug gets approved and is granted seven years of market exclusivity. If TKT's Replagal is running afoul of the FDA, that might just clear the path for Genzyme's Fabrazyme to claim the top prize.
Genzyme shares were up $2.70, or 13%, to $23.70.
Painful
Wednesday, TKT said the FDA's review of its approval application for
Replagal listed concerns with clinical data. In particular, the company
said regulators concluded that the data supporting the primary endpoint of
the drug's pivotal study -- a reduction in pain -- was not sufficient for
the drug's approval.
TKT says it will now try to use other data from its study in an attempt
to get Replagal approved, even tough the FDA also has concerns with this data. As a result, any FDA decision on the drug's
approval will be delayed into the first half of 2003, the company warned.
The company also said it is not yet sure when the FDA would schedule
another advisory panel meeting, although it hopes such a panel will meet at the end of the year or early next year.
What's got so many Wall Street followers of TKT so angry about this sudden change of fortune is that the company had not indicated that the FDA had problems with its data on Replagal.
Adam Feuerstein
RSS
