FDA panel backs Pfizer drug for rare genetic disease

NEW YORK — An experimental drug made by Pfizer for a rare and fatal neurodegenerative disease got a favorable vote from a Food and Drug Administration expert panel.

The FDA's Peripheral and Central Nervous System Drugs Advisory Committee voted 13-4 that the drug tafamidis meglumine would provide certain clinical benefits to patients with transthyretin familial amyloid polyneuropathy, or TTR-FAP, a hereditary disease that affects about 8,000 people worldwide. The FDA is not required to follow the recommendations of FDA advisory committees, but usually does.

The drug has received orphan drug designation, which the FDA gives to drugs that treat conditions affecting fewer than 200,000 people in the United States. Currently, there is no FDA-approved treatment for TTR-FAP.

"The panel's assessment represents a positive step forward in our goal to provide this much-needed medicine to patients suffering from this rare and fatal disease," Pfizer Specialty Care business unit medicines development group head Yvonne Greenstreet said. "Pfizer will continue to work with the FDA as the agency finalizes its review of our [new drug application] for tafamidis."


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