Pfizer Inc.’s (PFE) rare disease candidate, tafamidis meglumine, recently came up for review before the US Food and Drug Administration’s (FDA) Peripheral and Central Nervous System Drugs Advisory Committee. Pfizer is looking to get tafamidis approved for transthyretin familial amyloid polyneuropathy (TTR-FAP) in adult patients with symptomatic polyneuropathy to delay neurologic impairment.
The committee said that it did not find enough evidence of efficacy on a clinical endpoint. However, the committee voted 13-4 saying there was enough evidence to show efficacy on a surrogate endpoint which could predict a clinical benefit.
While the FDA is not required to follow the panel’s recommendation, it usually does so.
Tafamidis, which gained approval in the EU (trade name: Vyndaqel) in November 2011, faced a setback last year in the US when the FDA issued a “refusal to file” letter for the company’s NDA for the candidate. At that time, the FDA had said that the tafamidis NDA, which was submitted in February 2011, was incomplete.
The FDA accepted Pfizer’s resubmitted new drug application (NDA) for tafamidis in February 2012. Tafamidis became a part of Pfizer’s portfolio with its October 2010 acquisition of FoldRx. TTR-FAP is a progressively fatal genetic neurodegenerative disease.
Pfizer gained priority review status for tafamidis which means a response from the FDA should be out in June 2012. Tafamidis enjoys orphan drug status in the US. It is estimated that about 8,000 people across the world suffer from TTR-FAP. With tafamidis targeting a small patient population, we do not expect significant sales from the product.
We currently have a Neutral recommendation on Pfizer, which carries a Zacks #3 Rank (short-term Hold rating).
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