SpringWorks Therapeutics Secures FDA Approval For Second Cancer Drug

On Tuesday, the FDA approved SpringWorks Therapeutics, Inc.’s SWTX Gomekli (mirdametinib), a MEK inhibitor, for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.

With the approval, SpringWorks was granted a rare pediatric disease priority review voucher (PRV) by the FDA.

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NF1 is a genetic disorder affecting approximately 100,000 children and adults in the United States.

Patients with NF1 have an approximate 30-50% lifetime risk of developing plexiform neurofibromas (PNs), tumors that grow in an infiltrative pattern along the peripheral nerve sheath.

There are approximately 40,000 people in the United States living with NF1-PN.

The FDA approval was based on results from the Phase 2b ReNeu trial, which enrolled 114 patients with NF1-PN ≥2 years of age (58 adults and 56 pediatric patients).

Gomekli met the primary endpoint of confirmed objective response rate (ORR), demonstrating a 41% ORR (N= 24/ 58) in adults and 52% in children (N=29/56).

Tumor volume reductions were deep and durable; the median best percentage change in target PN volume was -41% in adults and -42% in children.

88% of adults and 90% of children with a confirmed response had a response of at least 12 months, and 50% and 48%, respectively, had a response of at least 24 months.

Patients in both cohorts also experienced early and sustained significant improvements from baseline in pain and quality of life.

Gomekli is available in 1 and 2 mg capsules and a 1mg tablet for oral suspension.

Gomekli is expected to be available within two weeks through a specialty pharmacy and specialty distributor network in the United States.

SpringWorks’ European marketing application for mirdametinib for children and adults with NF1-PN was validated by the European Medicines Agency and is currently under review; a decision is expected from the European Commission in 2025.

Price Action: SWTX stock was up 2.1% at $56.28 at last check Wednesday.

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