The FDA granted accelerated approval to Denali Therapeutics’ Avlayah (tividenofusp alfa) for Hunter syndrome, a decision that Denali frames as a return to a rare-disease surrogate-endpoint pathway after the agency’s recent string of tougher calls. Denali had faced scrutiny after prior setbacks, including a separate FDA rejection of Regenxbio’s related Hunter gene-therapy effort. Separately, the FDA cleared Corcept Therapeutics’ Lifyorli (relacorilant) in an accelerated timeline for ovarian cancer, adding another rare-disease- and oncology-relevant signal that the agency continues to move when evidence packages meet its threshold—even as it heightens review expectations in other cases. A parallel story emphasizes the broader impact on rare disease biotechs: FDA decisions now appear tightly linked to the quality and quantity of clinical evidence supporting both benefits and confirmatory pathways, with companies racing to align trial designs to FDA’s current evidentiary bar. Across the two approvals, market attention is likely to refocus on companies with accelerated-approval-ready endpoints and clear post-marketing study plans, especially where regulators previously rejected or delayed similar programs.