STAMFORD, Conn. - SpringWorks Therapeutics, Inc. (NASDAQ:SWTX) has commenced a rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for their investigational drug, mirdametinib. This MEK inhibitor is targeted for treating neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) in both pediatric and adult patients.
The submission is supported by data from the Phase 2b ReNeu trial, which demonstrated a 52% objective response rate in pediatric patients and 41% in adult patients, as of the data cutoff date of September 20, 2023. The trial, which enrolled 114 patients, indicated that mirdametinib could significantly improve pain, quality of life, and physical function, according to patient-reported outcome measures.
Mirdametinib has been generally well-tolerated, with the majority of adverse events being Grade 1 or Grade 2, including rash, diarrhea, and vomiting in pediatric patients, and rash, diarrhea, and nausea in adult patients. The drug has received Orphan Drug designation from both the FDA and the European Commission for the treatment of NF1, as well as Fast Track designation from the FDA for treating patients two years and older with NF1-PN that are progressing or causing significant morbidity. Additionally, in July 2023, mirdametinib was granted Rare Pediatric Disease designation by the FDA, which could lead to a priority review voucher upon approval.
SpringWorks expects to complete the NDA submission process in the second quarter of 2024. The company's CEO, Saqib Islam, expressed optimism about the potential of mirdametinib to become a best-in-class therapy for NF1-PN patients. He highlighted the company's commitment to working closely with the FDA during the review process.
Neurofibromatosis type 1 is a rare genetic disorder characterized by the growth of tumors along the nerves, which can lead to pain, disfigurement, and functional impairment. The ReNeu trial is ongoing, and it aims to further assess the efficacy, safety, and tolerability of mirdametinib.
This news is based on a press release statement from SpringWorks Therapeutics, Inc.
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