The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for an oral formulation of edaravone (MT-1186) for the treatment of amyotrophic lateral sclerosis (ALS).
Oral edaravone was developed to have a similar clinical profile as the intravenous (IV) formulation Radicava, which received FDA approval for ALS in May 2017. The exact mechanism by which edaravone exerts its therapeutic effect in patients with ALS is unknown.
The NDA submission includes several phase 1 clinical pharmacology studies comparing the oral suspension to the IV formulation. Additionally, results from a global open-label phase 3 study (ClinicalTrials.gov Identifier: NCT04165824) evaluating the safety and tolerability of edaravone oral suspension in 185 ALS patients showed a favorable safety profile.
“Our top priority is to help ensure ALS patients have flexibility with treatment and formulation options that are right for their specific needs,” said Atsushi Fujimoto, President, Mitsubishi Tanabe Pharma America. “We look forward to working with the FDA in the coming months to bring this new formulation to patients as soon as possible.”
A Prescription Drug User Fee Act (PDUFA) target date of May 12, 2022 has been set for the application.
- Mitsubishi Tanabe Pharma America announces FDA acceptance of New Drug Application (NDA) for oral edaravone formulation for the treatment of ALS. News release. Mitsubishi Tanabe Pharma America, Inc. Accessed January 12, 2022. https://www.prnewswire.com/news-releases/mitsubishi-tanabe-pharma-america-announces-fda-acceptance-of-new-drug-application-nda-for-oral-edaravone-formulation-for-the-treatment-of-als-301459460.html.
- Mitsubishi Tanabe Pharma America announces results from the global phase 3 safety study of investigational oral edaravone in the treatment of ALS. News release. Mitsubishi Tanabe Pharma America, Inc. December 9, 2021. Accessed January 12, 2022. https://www.mt-pharma-america.com/2021/12/09/mitsubishi-tanabe-pharma-announces-results-from-the-global-phase-3-safety-study/.
- Radicava. Package insert. Mitsubishi Tanabe Pharma America; 2021. Accessed January 12, 2022. https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/209176s010lbl.pdf.