Faculty efforts lead to orphan IV alternative for anti-seizure medication

An intravenous (IV) formulation created by faculty at the College of Pharmacy for topiramate, an antiseizure medication used to treat epilepsy, was submitted via a New Drug Application (NDA) to the Food and Drug Administration (FDA) in October 2025 and is pending review this year. The company developing the formulation is CURx.

Topiramate is an oral medication that currently lacks a FDA-approved IV formulation. If for any reason a patient faces an obstacle to taking the drug orally, they may be vulnerable to seizures. In a scenario like this, a patient might be placed on a different antiseizure medication that has an IV formulation, but its efficacy in that particular patient would be unknown, said Dr. James Cloyd, professor in the Department of Experimental and Clinical Pharmacology (ECP) and director of the Center for Orphan Drug Research (CODR) at the University of Minnesota.  

“Say you're a person with epilepsy placed on topiramate and you become seizure free. But then something happens that prevents you from taking the medication by mouth…for example, you have gastrointestinal surgery or you're in a coma,” Cloyd said. “Typically, you might be placed on another antiseizure med that has an IV formulation— the problem is you don't know if that other medication is going to be safe and effective, so you're taking a chance. What would be better is to replace the oral [medication] with the same drug administered IV.”

Because topiramate has multiple mechanisms of action, it’s also an attractive candidate to treat status epilepticus, a medical emergency in which a seizure lasts five minutes or longer, as well as neonatal seizures, or seizures that occur during a newborn child’s first month of life, Cloyd said. Both scenarios require rapid administration of a relatively large dose of a drug, called a loading dose.  

“We're hoping that IV topiramate will have expanded use in these and other conditions,” Cloyd said. “[Dr. Lisa Coles], a colleague in the ECP department and CODR, has been instrumental in carrying out modeling and simulations using data from studies conducted by our group as well as CURx to determine what kind of doses would be needed for seizure emergencies and as replacement for oral topiramate therapy.”

The IV topiramate formulation was originally created through a study at the college examining how the body’s absorption of epilepsy drugs (including topiramate) changes with age. This type of study requires an IV formulation to allow for comparison with the medication taken orally. Following the study, researchers at the college recognized that the IV formulation could address an unmet need for patients taking topiramate by offering them an alternative, or “replacement therapy,” should they be unable to take the medication by mouth. Based on studies in patients and healthy volunteers conducted by pharmacy and medical school faculty, the University of Minnesota licensed the new drug formulation to Ligand Pharmaceuticals, which then sub-licensed it to a smaller company called CURx Pharmaceuticals, Cloyd explained. He and his colleagues also applied for an orphan product designation, stating that the need for this drug formulation was in a small population— patients in whom oral topiramate therapy is interrupted. 

“The FDA approved the application, making IV topiramate an orphan product. The designation will provide the company with certain benefits, including seven years of U.S. market exclusivity, 50% tax credits on clinical testing costs, and exemption from FDA user fees.” Cloyd said. “The review will take place in 2026… we're cautiously optimistic the FDA will approve marketing of this drug.”