What parents of children with birth defects should know about Topamax

Topamax (generic name “topiramate”) is one of a class of medications known as “anticonvulsants.” According to the approved FDA label, Topamax is “indicated for” meaning approved for use by the FDA for:

• 1. Epilepsy – for seizure control
• 2. Lennox-Gastaut syndrome – to control seizures caused by this syndrome (approved 2001)
• 3. Prevention of migraine headaches – prevention only, not to relieve pain of migraine headaches when they occur

In the real world, Topamax (topiramate) is also used “off label” meaning doctors prescribe the drug for uses not approved by the FDA. This is legal. However, it is not legal for the manufacturer to promote sales of the drug (for example, advertising) for these “off label uses.” In fact, in May 2010, Ortho-McNeil Pharmaceutical LLC pleaded guilty to illegal promotion of Topamax and was sentenced to pay a criminal fine of over $6 million. Topamax has been used for neuropathic pain, anger control, post-traumatic stress disorder (PTSD) associated nightmares, metabolic diseases like adiposopathy, self injurious skin pricking, essential tremor, cluster headaches, and alcoholism.

Before March 2011, Topamax (topiramate) was a pregnancy category C drug. This means that studies on animals suggested risks to the fetus in human pregnancy but there was not enough information from human studies to prove an increased risk in humans. However, these animal studies are a signal to the manufacturer that there may be a danger of birth defects if Topamax is given to pregnant women. Once a drug is on the market, the manufacturing company has the primary responsibility to do follow up studies on signals of risks. Physicians are cautioned about the use of Pregnancy Category C drugs in the former label by suggesting Topamax should not be used in pregnancy unless benefits outweigh the potential risks. Unfortunately the manufacturer did not provide any data in humans on what that risk was. If there was a failure to warn children who suffered cleft palate and cleft lip and their families may have the right to bring a claim for the defects and the cost and suffering associated with the surgeries necessary to repair the problems. Families with an interest should contact an experienced attorney who is investigating this kind of case.

In March 2011, the FDA changed the Topamax (topiramate) to a pregnancy category D, reporting that data from registiries indicates infants exposed to Topamax before birth had an increased risk for cleft lip and cleft palate also known as oral clefts. The FDA approved label now warns doctors to consider the risks and benefits of Topamax before prescribing it for women of childbearing age, especially if the prescription is for a condition not usually associated with permanent injury or death (for example, migraine headaches). The new information for doctors gave the increased risks in humans so doctors could now assess the risks. For oral clefts, the increased risk was more than 3 times the background rate for women taking other antiepilepsy medications or over 300% (1.4% versus 0.38% – 0.55%) and over 20 times the background rate of mothers who were not taking epilepsy treatments. (1.4% versus 0.07%). There is another registry in England which showed very similar results.