Pfizer Receives 'Refuse to File' Letter from FDA
NEW YORK (AP) — Federal regulators have rejected Pfizer's application for a potentially fatal neurodegenerative disease, the drugmaker said Monday.
The Food and Drug Administration called the application for the drug, tafamidis, incomplete.
Pfizer plans to resubmit the application as quickly as possible, and it believes the additional information needed is available without conducting more clinical studies. The FDA sent Pfizer a "refusal to file" letter, which does not comment on the acceptability of the clinical data behind the drug or its effectiveness or safety.
Tafamidis is an oral treatment for patients with transthyretin familial amyloid polyneuropathy, a genetic neurodegenerative disease. Liver transplants are currently the only treatment option, and the condition affects about 8,000 people worldwide.
Pfizer, the world's largest drugmaker, acquired the compound last fall when it bought FoldRx Pharmaceuticals Inc., a privately held drug developer based in Cambridge, Mass.