* FDA seeks data from second trial, additional information
* Drug already approved in Europe under name Vyndaqel
June 18 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
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.