SpringWorks Therapeutics is a commercial-stage biopharmaceutical company focused on rare diseases and targeted oncology. SpringWorks sells two FDA-approved oral small molecule drugs for rare, genetically defined tumors. OGSIVEO (nirogacestat) is a gamma secretase inhibitor approved in November 2023 as the first and only treatment for desmoid tumors — rare, locally aggressive soft tissue tumors. GOMEKLI (mirdametinib) is a MEK inhibitor approved in February 2025 for adults and children with NF1-associated plexiform neurofibromas; it is the only approved therapy for adult NF1 patients and competes with AstraZeneca's Koselugo in the pediatric segment. Both drugs were originally licensed from Pfizer, and SpringWorks owes Pfizer tiered royalties on net sales plus commercial milestone payments. SpringWorks sells through specialty pharmacies and distributors, reaching specialist physicians with targeted sales forces of roughly 35 reps per product. Revenue grows as new patients start therapy and persist on treatment — both diseases are chronic, supporting long treatment durations. SpringWorks is pursuing EU and Japan approvals for both products. The pipeline includes nirogacestat in ovarian granulosa cell tumors (Phase 2 data expected in 2025), nirogacestat combinations with BCMA-directed agents in multiple myeloma, mirdametinib in pediatric low-grade glioma, and early-stage programs in TEAD inhibition and PP2A activation.
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