The FDA has granted a priority review designation to perampanel for 2 potential new indications for pediatric patients with seizures.
Lynn Kramer, MD
The FDA has granted a priority review designation to perampanel (Fycompa) for 2 potential new indications for pediatric patients with seizures, according to Eisai, the company developing the first-in-class AMPA receptor antagonist.
The first indication being reviewed by the FDA as part of a supplemental new drug application (sNDA) is for single-agent or adjunctive treatment for patients with partial-onset seizures (POS) with or without secondarily generalized seizures. The second indication is for perampanel monotherapy and adjunctive use for primary generalized tonic-clonic seizures (PGTC) in children with epilepsy.
"The FDA's Priority Review designation for this sNDA underscores the urgent need for more treatment options like Fycompa in the pediatric population," Lynn Kramer, MD, chief clinical officer and chief medical officer, Neurology Business Group, Eisai, said in a statement. "We are committed to working with the FDA to bring forward more treatments for young patients to help physicians achieve seizure freedom, a critical goal in epilepsy care."
The application was based on findings from 2 ongoing studies known as Study 311 and Study 232, and was made possible by a draft guidance issued by the FDA to speed up the development of new agents for neurologic disorders. FDA commissioner Scott Gottlieb, MD, labeled the guidance "a significant advance in the development of treatments for partial onset seizures in children."
"The guidance describes certain situations in which independent efficacy trials in pediatric patients are unnecessary and endorses a rigorous approach based on the extrapolation of effectiveness demonstrated in adult patients," he said. "The aim is to make the development of safe and effective treatments for pediatric patients suffering from these disorders more efficient."
Perampanel was initially approved in 2012 as an adjunctive therapy for POS. In 2015, this indication was expanded to include patients with PGTC, and again in 2017 the agent received an indication as a monotherapy for those with POS. In addition to the initial tablet version, an oral suspension formulation was also approved in 2016. The pediatric sNDA is for both formulations of the medication.
In the open-label Study 311 trial, perampanel oral suspension was given as an adjunctive therapy to children ages 4 to 11 with inadequately controlled POS or PGTC seizures. The study included 149 children with POS and 31 with PGTC seizures. The overall mean daily dose of the medication was 6 mg.
A second open-label trial, Study 232, evaluated pharmacokinetics with perampanel. In this trial, which also sought to uncover preliminary safety and efficacy data, perampanel was given as an adjunctive therapy in pediatric patients aged 2 to less than 12 years with epilepsy.
Although efficacy data were not yet available, the FDA noted in its new guidance that findings in adults seem to be similar to those in children for POS, allowing for adult treatments to expand to pediatric use.
"The incidence of seizures in the pediatric age group has been steadily increasing in the US where an estimated 470,000 children are living with epilepsy today. Approximately 30% of all patients have uncontrolled seizures, and it is crucial that we do whatever we can to help these patients achieve the ultimate goal of seizure freedom," said Trevor Resnick, MD, Pediatric Neurologist at Nicklaus Children's Hospital.
"Approximately 71% of all people living with epilepsy miss at least one dose of their medication per month, and 45% of those missed doses result in a subsequent breakthrough seizure. Fycompa's long, 105-hour half-life may play an important role for patients who miss doses," said Resnick.
Under the priority designation, the FDA will review the sNDA for pediatric seizures within 6 months from the acceptance of the filing compared with the standard 10 months, according to the Prescription Drug User Fee Act. Under this timeline, the FDA is likely to make its decision in December 2018.