The FDA has committed to unprecedented transparency by publicly releasing complete response letters (CRLs) in real time upon issuance, alongside disclosures of previously unpublished CRLs related to both approved and ongoing drug applications. This move aims to bolster public trust, expedite communication with stakeholders, and clarify regulatory decisions. Meanwhile, the agency prepares a new flexible review pathway for ultra-rare genetic diseases, reflecting an adaptive regulatory environment responsive to emerging therapeutic modalities.