FDA panel positive on Genzyme drug
Panel's move could open door for final FDA approval
By Ted Griffith, CBS.MarketWatch.com
Last Update: 8:13 PM ET Jan. 13, 2003
BETHESDA, Md. (CBS.MW) -- In a boost to Genzyme's bid to win approval for a treatment for the rare Fabry disease, an influential FDA advisory panel has concluded the biotech firm's drug met an important effectiveness goal in clinical testing.
The panel late Monday voted 14-1 to advise the FDA that Genzyme's Fabrazyme drug reduced fatty deposits in the blood vessels of the kidneys, a key measure of the therapy's effectiveness.
Shares of Genzyme (GENZ: news, chart, profile) were halted Monday. On Friday, shares surged $2.67, or 9 percent, to $31.58.
The panel's move bolsters Genzyme's drive to become the first company in the United States to market a treatment for Fabry disease, a rare genetic illness. The FDA typically relies heavily on the advice of its panels, which are made up of experts in the relevant medical specialties.
"We're very encouraged by the panel's vote," said Genzyme spokesman Dan Quinn. "This endorses the approach we took in clinical trials and the success we had in meeting our primary [effectiveness goal]."
A final decision on whether to approve the Fabrazyme drug is expected in the first half of the year.
The panel's findings may be bad news for Transkaryotic Therapies, a rival biotech firm that's also hoping to market a Fabry disease treatment in the United States. Both companies' drugs are approved for sale in Europe.
Transkaryotic's drug, Replagal, is scheduled to be reviewed by the advisory panel Tuesday. FDA staff members, in a commentary posted on the agency's Web site Monday, said Transkaryotic's drug fell short of effectiveness goals related to improvement in kidney and heart function.
Transkaryotic, known as TKT, has previously disclosed that, based on preliminary data, Replagal didn't meet its primary effectiveness goal in a round of late-stage patient testing.
Shares of TKT (TKTX: news, chart, profile) were halted Monday at $8.74.
Patients with Fabry disease lack an enzyme to break down fat, which increases the risk of organ failure and strokes. Investment firm SG Cowen has estimated there are about 2,000 patients with Fabry disease in the United States.
Ted Griffith is a reporter for CBS.MarketWatch.com
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