Ultragenyx’s latest Sanfilippo approval arrives alongside reports that the therapy will cost $4 million per patient, adding another high-priced gene therapy to the US market. The company’s second gene therapy approval since August strengthens its commercial position and raises the stakes for rare-disease access conversations. Separately, the same reporting roundup also flagged Bristol Myers Squibb’s decision to shelve Orum, illustrating how companies manage portfolios as clinical and commercial signals shift. For payers and health systems, the price underscores the need for outcomes-based contracting and strict patient selection as the gene therapy pipeline keeps expanding.