FDA Tightens Accelerated Approval Pathway, Rejects Regenxbio and Disc Medicine
The Food and Drug Administration review process for accelerated approvals was meant to drugs for rare diseases to pass the approval process, clearing the drugs based on early biological signs instead of waiting years for definitive proof of real-world outcomes, David Wainer of The Wall Street Journal. Now, however, the accelerated approval pathway isn't closed but has become much narrower and harder to predict. Recently, the FDA issued a string of rejections, including Regenxbio's (RGNX) gene therapy for Hunter Syndrome and Disc Medicine's (IRON) treatment for a blood disorder. In some cases, the agency reversed its own guidelines provided to the companies. In 2025, the FDA greenlighted only nine accelerated approvals, down from 20 in 2024. Other companies in the space include: uniQure (QURE), Moderna (MRNA), Replimune (REPL), and Capricor (CAPR).
