Mirum Wins FOP Approval Despite Primary Endpoint Miss & Carvykti's Five-Year Data

Mirum's Atebrioz (zilurgisertib) wins FDA approval for FOP despite a primary-endpoint miss; Carvykti (ciltacabtagene autoleucel) shows five-year disease control without maintenance in half of 20 myeloma patients. aTyr plans another efzofitimod trial in sarcoidosis. The roundup covers Pharming's Joenja (leniolisib), ADARx's IPO, Canada's Epkinly (epcoritamab) expansion, Novo–Nanexa's long-acting peptide deal, FDA nominee Heidi Overton's hearing, and Medicaid's GENEROUS pricing plan. September 25 edition, published September 26: relative dates and pending events reflect the original reporting cutoff.Alex Mercer and Maya Patel host. Get the daily rundown in your inbox — subscribe at https://thepharmacloseout.com.