FDA Panel Sets Fabry Review for January

An advisory panel of the Food and Drug Administration will review the respective Fabry disease drugs of Genzyme General(GENZ Quote - Cramer on GENZ - Stock Picks) and Transkaryotic Therapies(TKTX Quote - Cramer on TKTX - Stock Picks) in January, the companies said Monday.

The advisory panel meeting, scheduled for Jan. 13-14, comes after a similar review in September was cancelled due to conflict of interest problems were raised about certain panel members.

Earlier this month, Transkaryotic shares plunged after the biotech firm disclosed that U.S. drug regulators refused to accept certain data supporting the efficacy of its drug, Replagal. The company has insisted that alternative data will be sufficient for the FDA to approve its drug.

Genzyme's drug is known as Fabrazyme.

The recommendations of the advisory panel will be sent to the FDA for a final ruling. The agency is supposed to render a final decision under rules defined by the Orphan Drug Act, which means that only one drug gets approved and is given seven years of market exclusivity in the U.S.

In its statement, Transkaryotic officials said they expect the FDA to issue a final decision during the first half of 2003.

Fabry disease is rare -- only about 2,000 patients suffer from it in the U.S. -- but treatment is expected to cost upwards of $165,000 per year, which makes this a $330 million market opportunity for both companies.