The US Food and Drug Administration's (FDA) Peripheral and Central Nervous System Drugs Advisory Committee voted on 24 May to partially support Pfizer's drug, tafamadis, which is looking for FDA approval to treat a rare neurodegenerative disease.

The panel voted 13 to 4 against the drug in an initial vote, saying study data did not show the drug to be effective in directly slowing the progression of familial amyloid polyneuropathy (FAP). The committee later voted again, finding in a 13 to 4 decision that tafamadis was effective in treating surrogate endpoints associated with FAP such as limb function.

The split vote may spell trouble of tafamadis at FDA, reports Reuters, as its staff "recommended rejecting the drug saying the data did not prove that it worked well in treating the disease." Those concerns are likely to be weighed against the needs of the approximately 2,500 US patients suffering from FAP who currently lack medicinal options to treat the disorder, reports Med Page Today.

The drug is already approved by the European Medicines Agency (EMA) for the condition, and is marketed under the trade name Vyndaqel.

Read more:

Reuters - U.S. panel split on Pfizer rare disease drug

Med Page Today - FDA Panel Sends Mixed Message on Pfizer Drug

PMLiVE - FDA panel supports Pfizer's tafamidis despite efficacy concerns

The Pink Sheet Daily - Pfizer's Vyndaqel Splits Advisory Committee; Now It Must Move FDA