The U.S. Food and Drug Administration has two PDUFA dates this week, one for a type of epilepsy and the other for a triple-combination drug for cystic fibrosis. Here’s a look.
The U.S. Food and Drug Administration (FDA) has two PDUFA dates this week, one for a type of epilepsy and the other for a triple-combination drug for cystic fibrosis. Here’s a look.
Eton Pharmaceuticals’ Lamotrigine for Seizures
Eton Pharmaceuticals, based in Deer Park, Illinois, has a target action date of March 17, 2020 for its New Drug Application for ET-105, its innovative formulation of lamotrigine. Eton acquired the U.S. marketing rights to the drug in June 2019 from Aucta Pharmaceuticals. The company is seeking approval for the drug as an adjunct therapy for partial seizures, primary generalized tonic-clonic seizures, and generalized seizures of Lennox-Gastaut syndrome in patients two years of age and older.
Lennox-Gastaut syndrome is a severe condition marked by epilepsy that begins early in life. There are multiple types of seizures, a specific pattern of brain activity known as slow spike-and-wave measured by EEG, and impaired mental abilities. The seizures typically begin between the ages of three and five.
Lamotrigine is a widely used drug in the U.S for seizures. ET-105’s innovative formula is an oral liquid specifically designed to treat patients with dysphagia, which is difficulty swallowing. Lamotrigine is currently available in tablet form and usually at doses stronger than needed for this patient population.
At its annual report on March 5, the company stated, “As previously disclosed, Eton received comments from the FDA requesting changes to the Dosage and Administration section of the product’s Prescribing Information to simplify the dosing information for intended users, and the FDA requested a human factors validation study with the revised labeling to demonstrate that the intended users can accurately prepare and administer the oral suspension. As a result, Eton expects to receive a CRL on its PDUFA date of March 17, 2020. The company has begun drafting the human factors study protocol and expects to have the study completed and resubmitted to the FDA in the second or third quarter of this year, which would allow for final approval of the NDA before the year end. During the quarter, Eton’s partner was granted a patent by the United States Patent and Trademark Office for ET-105’s unique formulation. Eton expects the patent to be Orange Book listed after product approval.”
Vertex Pharmaceuticals’ Elexacaftor Triple Combo for Cystic Fibrosis
Boston-based Vertex Pharmaceuticals has a target action date of March 19 for its NDA for VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination. The submission was based on positive data from two global Phase III trials in patients with cystic fibrosis—a 24-week Phase III trial in people with one F508del mutation and one minimal function mutation and a four-week Phase III trial in patients with two F508del mutations. Both trials demonstrated statistically significant improvements in lung function, the primary endpoint, and in all key secondary endpoints.
“If approved, the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen would be a significant advance in CF treatment as the first CFTR modulator for those with one F508del mutation and one minimal function mutation, and bring additional benefit to patients with two F508del mutations,” said Reshma Kewalramani, executive vice president and chief medical officer of Vertex, in August 2019 when the NDA submission was accepted. “Our goal is to provide medicines that treat the underlying cause of CF to the vast majority of people with CF. We share a sense of urgency with people with CF, caregivers and clinicians to rapidly deliver innovative CF medicines to those waiting, and we look forward to working with the agency as they review the application over the course of the coming months.”
