FDA now accepts Pfizer's tafamidis NDA

Having around a year ago issued a refuse to file letter (The Pharma Letter April 5, 2011), the US Food and Drug Administration has accepted for review global drugs behemoth Pfizer's (NYSE: PFE) New Drug Application for tafamidis meglumine, the company's investigational, novel, oral therapy for the treatment of Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP).

TTR-FAP is a rare, progressive and fatal neurodegenerative disease that affects approximately 8,000 patients worldwide. The European Commission approved tafamidis, the trade name of which is Vyndaqel in the European Union, last fall (TPL November 18, 2011). The drug came to Pfizer along with its acquisition of FoldRx Pharmaceuticals to expand its rare disease portfolio (TPL: September 2, 2010).

The FDA has granted the tafamidis NDA a priority-review designation and has provided an anticipated Prescription Drug User Fee Act (PDUFA) action date in June 2012. Priority-review designation is granted to drugs that have the potential to offer significant improvement in treatment or provide a treatment where no adequate therapy exists.