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US panel split on Pfizer drug for familial amyloid polyneuropathy

(Reuters) – A U.S. health advisory panel on Thursday issued a split vote on data for Pfizer Inc’s drug tafamidis to treat familial amyloid polyneuropathy, a rare disease that affects as many as 10,000 people worldwide, including about 2,500 Americans.

The U.S. Food and Drug Administration panel voted 13 to 4 that the drug did not show that it met the main goal in a study in treating the fatal condition.

However, the panel also voted 13 to 4 that the drug treated a surrogate endpoint, which may correlate with treating the underlying disease.

The panel’s recommendation will be considered by the U.S Food and Drug Administration when it makes a decision on tafamidis.

Tafamidis is already approved in Europe under the name Vyndaqel.

FDA staff on Tuesday recommended rejecting the drug saying the data did not prove that it worked well in treating the disease.

Most people with the genetic mutation that is thought to be the most common cause of the disease live in Portugal, Sweden or Japan. Once symptoms begin, a majority of people die in 11 years, the FDA said. The only current treatment for the disease is a liver transplant.

Two FDA reviewers said during a clinical trial that tafamidis was most successful in treating people in Portugal, but it was unclear why, and 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.