Topiramate reclassified as Category D due to potential link to oral clefts in newborns

FDA announced that it was reclassifying topiramate from a Pregnancy Category C to a Pregnancy Category D drug.

Key Points

FDA announced that it was reclassifying topiramate (Topamax, Ortho-McNeil-Janssen Pharmaceuticals) from a Pregnancy Category C to a Pregnancy Category D drug. This decision was based upon newly available data in humans suggesting a link between the drug's use in the first trimester of pregnancy and oral cleft (a small notch in the lip or a groove that runs into the roof of the mouth and nose) formation in newborns.

Pregnancy Category D drugs are those with positive evidence of human fetal risk based on human data, but still may be used in pregnant women in certain situations when its benefits are thought to outweigh potential risks. Previously, data suggesting fetal harm with topiramate was available only from animal studies.

Topiramate first gained FDA marketing approval in 1996 and is currently indicated for the treatment of certain types of seizure disorders and prevention of migraine. Topiramate also is frequently used for various off-label purposes (eg, treatment of psychiatric disorders, neuropathic pain, and substance abuse).

In their published safety communication, FDA agency officials stressed, "The benefits and the risks of topiramate should be carefully weighed when prescribing this drug for women of childbearing age, particularly when topiramate is considered for a condition not usually associated with permanent injury or death." They continued, "Appropriate alternative treatment should be considered."

Because evidence suggested oral clefts form during the first trimester of pregnancy-before many women know they are pregnant-the agency is also recommending prescribers encourage the use of effective forms of contraception in women of childbearing age prescribed topiramate.

It is estimated that about 4.3 million Americans filled at least 1 topiramate prescription at a retail pharmacy between 2007 and 2010.


FDA. FDA Drug Safety Communication: Risk of oral clefts in children born to mothers taking topiramate (Topamax). Available at: Accessed March 5, 2011.

Topamax [package insert]. Titusville, NJ: Ortho-McNeil-Janssen Pharmaceuticals, Inc.; 2009.

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