May 22, 2012 / 1:27 PM / 6 years ago

FDA staff shoots down Pfizer rare disease drug

WASHINGTON (Reuters) - Drug reviewers on Tuesday recommended rejecting a Pfizer Inc drug because the data did not prove it worked well in treating a rare neurodegenerative disease.

People walk past the Pfizer World headquaters in New York, February 3, 2010. REUTERS/Brendan McDermid

The U.S. Food and Drug Administration staff reviewed the pill, tafamidis, ahead of an advisory panel of outside experts, which will vote on whether to recommend it for approval on Thursday. The FDA will make a final decision later, taking into account the panel’s recommendation.

Tafamidis, which is already approved in Europe under the name Vyndaqel, is meant to treat familial amyloid polyneuropathy, a fatal condition that affects as many as 10,000 people worldwide, including about 2,500 Americans.

Most people with the genetic mutation that is thought to cause the disease live in Portugal, Sweden or Japan. Once symptoms begin, most people die in 11 years, the FDA said. The only current treatment for the disease is a liver transplant, which keeps 80 percent of patients alive after five years.

Two FDA reviewers said during a clinical trial that tafamidis was most successful in treating people in Portugal, but the data did not prove it helped those with the disease in other locations.

People taking tafamidis during clinical trials also appeared to have a milder disease than those taking a placebo, which could skew the results, they said.

Reporting by Anna Yukhananov; Editing by Maureen Bavdek; Editing by Gerald E. McCormick

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