Transkaryotic Warns of More FDA Concerns

Transkaryotic Therapies Inc. Chief Executive Richard Selden said U.S. regulators might have more extensive concerns about its experimental Replagal medicine for Fabry disease than the company indicated last week.

The biotechnology company is trying to win U.S. clearance of Replagal for conditions related to a rare genetic disorder that destroys major organs and causes severe pain. Transkaryotic has said the Food and Drug Administration found the company’s pain-reduction study results insufficient to support approval.

The company said last week that it would pursue Replagal clearance based on research evaluating whether the drug helps protect kidney and heart function, a secondary goal of its studies. Selden said Tuesday that the FDA disagrees with Transkaryotic’s interpretation of those findings, though the company expects to have a chance to persuade regulators at an FDA panel hearing this year or early 2003.