FDA approval of Isembyld gives spinal muscular atrophy its first muscle-targeted therapy, moving motor function in patients plateaued on Spinraza or Evrysdi while introducing a 9% fracture warning and a double-biologic payer burden.
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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.
AbbVie's etentamig beat older salvage on response and progression-free survival in Phase 3 CERVINO. The harder question is whether a single step-up and monthly intravenous schedule can loosen the REMS-and-hospitalization architecture that still gates approved BCMA T-cell engagers.
uniQure asked FDA for accelerated approval of AMT-130 on a 12-patient, 36-month high-dose dataset, after the agency twice reversed itself on external-control evidence.