IntraBio’s Aqneursa cleared the FDA as the first treatment for ataxia-telangiectasia on a -1.88 SARA point crossover win, treating downstream neurodegeneration rather than an intractable 305 kDa kinase defect.
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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.
The FDA has granted accelerated approval to Bayer's Hyrnuo as a frontline therapy in HER2-mutant lung cancer, matching rival zongertinib's response rates while carrying twice-daily meal requirements and steeper gastrointestinal toxicity.
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.