SpringWorks introduces oral innovative MEK inhibitor from Pfizer Inc. (PFE.US) approved by FDA for market launch.

On February 11th local time, SpringWorks Therapeutics announced that the US FDA has approved its MEK inhibitor Gomekli (mirdametinib) for the treatment of symptomatic plexiform neurofibromas (PN) in patients with type 1 neurofibromatosis (NF1) who are 2 years and older and cannot be completely removed. The press release stated that this is the first and only drug approved for the treatment of NF1-PN in adults and children. The product was acquired by SpringWorks from Pfizer Inc. (PFE.US). The approval was based on the results of the Phase IIb ReNeu trial, which enrolled 114 NF1-PN patients aged 2 years (58 adults and 56 pediatric patients). The data showed that Gomekli achieved the primary endpoint of objective response rate (ORR), with an ORR of 41% (24/58) in adult patients and 52% (29/56) in pediatric patients. Tumor volumes in treated patients significantly decreased and persisted; the median percentage change in target PN volume was -41% in adult patients and -42% in pediatric patients. In adult and pediatric patients, 88% and 90% of patients, respectively, had a duration of response of at least 12 months; additionally, 50% and 48% of patients had a duration of response of at least 24 months. According to assessments using multiple patient-reported outcome tools, pain and quality of life significantly improved from baseline and continued to improve in both groups of patients.