UPDATE 1-FDA seeks more data on Pfizer rare disease drug

Tue Jun 19, 2012 3:32am IST

Related Topics

Stocks

   

* FDA seeks data from second trial, additional information

* Drug already approved in Europe under name Vyndaqel

June 18 (Reuters) - U.S. health regulators declined to approve a Pfizer Inc's drug for a rare and fatal neurodegenerative disease until a second study can establish the effectiveness of the treatment, the company said on Monday.

Pfizer said the Food and Drug Administration, in a so-called complete response letter, told the company it would not approve the drug, tafamidis meglumine, at this time. It asked for additional information on data already submitted to the agency and requested completion of a second efficacy study, the company said.

The drug, intended to treat a condition called transthyretin familial amyloid polyneuropathy (TTR-FAP), was approved for sale in Europe last November under the brand name Vyndaqel.

The FDA decision to seek more information comes after agency reviewers, ahead of an advisory panel meeting of outside experts in May, recommended the drug's rejection, saying data had not proved that tafamidis worked well enough.

The advisory panel then issued a split vote, saying that the drug failed to show it met the primary goal of a pivotal study, but that it was successful on another measure.

"It is our intention to request a meeting as soon as possible with the agency in order to discuss a potential path forward," Dr. Yvonne Greenstreet, head of the medicines development group for Pfizer's Specialty Care Business Unit, said in a statement.

TTR-FAP is a fatal genetic disease that affects about 8,000 patients worldwide and progressively destroys quality of life. As the disease advances, patients can lose the ability to walk, needing wheelchair assistance, and eventually become bedridden and unable to care for themselves.

The Pfizer drug is designed to delay neurologic impairment from the disease.

FILED UNDER: