With only about 5% of rare conditions carrying an FDA-approved treatment, the agency is trying to turn case-by-case flexibility into written, predictable defaults. Whether that produces certainty or new rigidity is the open question.
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KRAS sat on the undruggable list for decades. Silexion's SIL204 is built to silence the mutation earlier in the cell, and the Phase 2/3 program is now moving into patients.
PhRMA sued CMS on October 7 over the GLOBE most-favored-nation rule for Medicare Part B. The final rule covers as few as four manufacturers and $440 million in projected savings. The suit's real target is the CMMI test authority beneath it.
ArriVent's firmonertinib beat chemotherapy on investigator reads in a 398-patient Phase 3, but the blinded central review it chose returned p=0.0654, and first-line EGFR exon 20 lung cancer now belongs to combinations that keep chemotherapy in the regimen.