FDA denies Pfizer's bid for rare disease drug

U.S. regulators snubbed Pfizer's application for approval of the drug tafamidis meglumine, dealing the drug giant a setback in its quest to build its rare-disease drug business. The FDA shot down the company's bid with a Complete Response Letter, in which the agency asks for more data on the efficacy of the drug for transthyretin familial amyloid polyneuropathy (TTR-FAP), Pfizer ($PFE) said late Monday.

It's no surprise to see the agency hold back on green-lighting Pfizer's drug before a second study is done to show effectiveness. Agency staff had blasted the application last month, recommending a CRL after noting that a key trial failed to achieve co-primary endpoints and questioning the consistency of findings among 8 study sites. Non-FDA advisers gave the program a mixed review, voting 13-4 that Pfizer missed the mark in the study but, in a separate 13-4 vote, endorsing the potential of the therapy to provide clinical benefits to patients.

European regulators stamped an approval on tafamidis for TTR-FAP in November. But there are no available treatments for the disease in the U.S., a fact that Pfizer has repeated in its quest to deliver the first approved drug to an estimated 3,000 patients with the disorder in the country. Tafamidis is designed to stabilize the transthyretin protein, which falls apart in patients with TTR-FAP and causes defective proteins build up in the nerves and cause a range of nasty symptoms. Pfizer picked up the product in its 2010 buyout of the biotech group FoldRx.

"TTR-FAP is a relentless and debilitating disease. We understand the urgent need within the patient community and stand firmly behind this innovative medicine," said Dr. Yvonne Greenstreet, senior vice president and head of medicines development group for Pfizer's specialty care unit, in a statement. "It is our intention to request a meeting as soon as possible with the agency in order to discuss a potential path forward."

"I"ll tell you this about tafamadis," said Jose Carlos Gutiérrez-Ramos, head of biotherapeutics R&D at Pfizer, in an interview with FierceBiotech Editor-in-Chief John Carroll at the 2012 BIO International Convention. "It's a great drug and it will get to patients."

It appears, though, that they will have to wait.

- here's Pfizer's release
- check out Bloomberg's article