FDA accepts Pfizer application for genetic disease drug

Pfizer hoping for approval of drug to treat disorder that affects 8,000 people worldwide

NEW YORK — The Food and Drug Administration will review a regulatory approval application from Pfizer for a drug to treat an extremely rare and fatal degenerative disease, Pfizer said Wednesday.

Pfizer announced that the FDA had accepted its application for tafamidis meglumine, a treatment for transthyretin familial amyloid polyneuropathy. The disease, also known as TTR-FAP, is a fatal genetic illness that affects about 8,000 people worldwide and causes severe sensory loss, pain, weakness and organ dysfunction.

The FDA expected to reach a decision on the drug by June. The European Commission approved the drug in November 2011 under the brand name Vyndaqel.

 


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