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Pfizer Drug for Rare Disease Shouldn't Be Approved, US Review Says [UPDATE]


The big drug company needs to bring new products to market, but FDA staffers say a company study doesn't show that the treatment Vyndaqel works.


UPDATE, May 25, 2012:
In what can best be described as a split decision, a panel of US government advisers said that a study by Pfizer (PFE) failed to show a drug for a rare disease worked because it didn't meet the goal of the trial but the treatment may have merit based on other findings.

In a vote of 13-4, advisers to the Food and Drug Administration found that Pfizer didn't persuade them that the drug was statistically more effective in treating the disease than a placebo given to patients in a study. However, the advisers voted 13-4 that other study findings showed that there's reasonable evidence that the drug may be effective treating so-called surrogate endpoints. In other words, the drug may treat symptoms of the disease. The two votes presents a decision challenge for the FDA, which will have final say on the drug's approval. The agency isn't bound by its advisers' recommendations.

Pfizer will try to sway a panel of government advisers Thursday on the benefits of its drug Vyndaqel for treatment of a rare, inherited neurodegenerative disease. But it may be a long day for some company presenters.

In a harsh report on the company's drug, Food and Drug Administration staff reviewers said Tuesday that Pfizer is trying to win approval for a treatment that lacks an acceptable study showing the product works.

"I recommend a complete response action, based on inadequate evidence of effectiveness," says FDA reviewer Devanand Jillapalli in a document prepared for Thursday's advisory panel. Complete response is FDA jargon for a rejection.

Vyndaqel was approved for use in Europe last year and Pfizer wants to get clearance to sell the drug in the US. The drug is designed to treat a condition known as transthyretin familial amyloid polyneuropathy. There's no approved treatment for the deadly disease in the US. The only therapy for patients in this country is to receive a liver transplant.

As much as there's a need for a drug, FDA staff reviewers say they disagree with Pfizer's conclusion that a key company study showed Vyndaqel's effectiveness. In fact, the study has "important weaknesses," FDA reviewers say. The study didn't meet a target to show that it is effective from a statistically significant standpoint. Pfizer says the data still points to an effective drug.

How could Pfizer, an experienced drug developer, misread the FDA?

Pfizer says in its own briefing document that its clinical program was developed under a speedy review process allowed by the FDA and the agency was frequently advised.

In a statement to Minyanville, the company says, "Pfizer believes that the data for (the drug) provide substantial evidence of effectiveness and meaningful therapeutic benefit where there is a high unmet medical need."

The FDA staff report certainly isn't the final word. The advisers will consider the drug's benefits and make their own recommendation to the agency, which will decide whether to approve the treatment.

Pfizer desperately needs to bring new drugs to market as it loses patent protection on blockbusters such as cholesterol drug Lipitor, which is now subject to generic competition.

Twitter: @brettchase

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