The U.S. Food and Drug Administration (FDA) has approved the orphan drug, sotorasib (brand name: Lumakras), to treat adult patients with KRAS G12C-mutated non-small cell lung cancer and who have received at least one prior systemic therapy. 

This is the first approved targeted therapy for tumors with any KRAS mutation, which accounts for approximately 25% of mutations in non-small cell lung cancers. KRAS G12C mutations represent about 13% of mutations in non-small cell lung cancers. Prior to this approval, KRAS mutations were considered resistant to drug therapy. 

The FDA approval of sotorasib is largely based on results from a subset of patients in CodeBreaK 100, the largest clinical trial conducted exclusively for patients with the KRAS G12C mutation. The trial demonstrated favorable efficacy and tolerability in 124 patients with KRAS G12C mutation-positive non-small cell lung cancer who had disease progression after receiving an immunotherapy and/or chemotherapy. Sotorasib, when administered orally once daily, demonstrated an ORR of 36% (95% CI: 28-45) with 81% (95% CI: 73-87) of patients achieving disease control. The median duration of response was 10 months. The most common adverse reactions were diarrhea, musculoskeletal pain, nausea, fatigue, hepatotoxicity and cough. Adverse reactions resulting in permanent discontinuation of sotorasib occurred in 9% of patients.

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