The previously approved therapy is now indicated for treatment in pediatric patients age 2 to 10.
Darcy Krueger, MD, PhD
The FDA has approved an expanded indication for vigabatrin (Sabril; Lundbeck Pharmaceutical) for the treatment of refractory complex partial seizures (CPS) in patients 2 to 10 years old.1 Previously, the drug was approved for patients with refractory CPS who were age 10 and older.
CPS are typically experienced by patients with tuberous sclerosis complex (TSC), and often don’t respond to typical antiseizure medications.
“We are happy to learn the FDA updated the indication so TSC patients of any age can obtain the medication when recommended by their neurologists," Darcy Krueger, MD, PhD, director of the Cincinnati Children’s Hospital Medical Center TSC Clinic, said in a statement.1
Vigabatrin was originally approved in August 2009 as monotherapy for infantile spasms in patients age 1 month to 2 years and as adjunctive therapy for refractory CPS in patients age 10 and older.2 Ten years later, the FDA approved the first generic version of vigabatrin for the adjunctive treatment of CPS in patients with refractory epilepsy age 10 and older.3
The expanded FDA approval was based on data from 3 double-blind, placebo-controlled, parallel-group, adjunctive-treatment studies that evaluated 373 patients aged 3 to 16 with uncontrolled CPS with or without secondary generalization.
Pooled data from these studies showed a similar dose response relationship between adult and pediatric patients.
A supplemental study funded by the Tuberous Sclerosis Alliance (TS Alliance) was included in Lundbeck’s application to the FDA for the label expansion after the TS Alliance received multiple complaints from within the TSC community that children age 3 to 9 were being denied coverage for vigabatrin.
"This new FDA approval is a game changer for the TSC community, and we are incredibly grateful to our partners at Lundbeck who understood the need and worked diligently to obtain this label change for patients," Kari Luther Rosbeck, president and CEO of TS Alliance, said in a statement.1
Notably, vigabatrin was originally granted approval with a REMS (risk evaluation and mitigation strategy) due to its associated risk of vision loss and suicidal thoughts and behaviors. In 2016, the FDA approved a modified REMS that no longer requires the submission of ophthalmologic assessment forms, though these assessments should still occur periodically as described in the drug label.2
1. Tuberous Sclerosis Alliance lauds Lundbeck and FDA for Sabril label expansion for patients 2 to 10 years old [news release]. Silver Spring, MD. Tuberous Sclerosis Alliance. January 27, 2020. prnewswire.com/news-releases/tuberous-sclerosis-alliance-lauds-lundbeck-and-fda-for-sabril-label-expansion-for-patients-2-to-10-years-old-300993825.html. Accessed January 30, 2020.
2. Sabril (vigabatrin). FDA. fda.gov/drugs/postmarket-drug-safety-information-patients-and-providers/sabril-vigabatrin. Updated June 21, 2016. Accessed January 30, 2020.
3. FDA approves first generic version of Sabril to help treat seizures in adults and pediatric patients with epilepsy [news release]. FDA. January 16, 2019. fda.gov/news-events/press-announcements/fda-approves-first-generic-version-sabril-help-treat-seizures-adults-and-pediatric-patients-epilepsy. Accessed January 30, 2020.