Key Takeaways AGIO stock dropped 11% as the FDA extended Pyrukynd's thalassemia review by three months.The delay follows Agios' proposed REMS to address liver injury risks flagged in original filings.A decision is now expected on Dec. 7, 2025, instead of the original September target date.Shares of Agios Pharmaceuticals (AGIO) fell 11% on Thursday after the company announced that the FDA had extended the review timeline for the supplemental new drug application (sNDA) seeking label expansion for its sole ma ...

Drug Safety Review - The Health and Human Services Secretary requested a review of data on methapristone (abortion pill) [1] - The review is ongoing as part of a risk evaluation and mitigation strategy (REMS) commitment [2] - The review includes examining self-reported data in the adverse event reporting database [2] - The review also considers studies suggesting potential safety signals in large administrative databases, such as drug-drug interactions [3] Access to Medication - The company has no plans to change access to methapristone [3]

Group 1 - The FDA has extended the PDUFA action date for Cytokinetics Inc's aficamten to December 26 from September 26 due to the need for additional time to review the proposed REMS [1][3] - Cytokinetics submitted the NDA for aficamten without an accompanying REMS, but the FDA later requested a REMS based on the drug's characteristics [2] - The submission of the REMS is considered a Major Amendment to the NDA, resulting in a standard three-month extension [3] Group 2 - Cytokinetics' CEO expressed confidence in the benefit-risk profile of aficamten and anticipates a differentiated label upon potential FDA approval [4] - Bristol Myers Squibb's Camzyos did not meet its primary endpoints in a recent Phase 3 trial, which may create an opportunity for aficamten [5] - Analysts believe Cytokinetics is well-positioned to benefit from Bristol Myers' groundwork, with expectations for aficamten to secure a differentiated REMS [6]

Core Viewpoint - The FDA has extended the PDUFA action date for Cytokinetics' aficamten to December 26, 2025, due to the need for additional time to review the proposed REMS [1][2]. Group 1: FDA Review Process - Cytokinetics submitted the NDA for aficamten without an accompanying REMS, which was later requested by the FDA during the review process [2]. - The submission of the REMS is considered a Major Amendment to the NDA, resulting in a standard three-month extension to the original PDUFA action date [2]. Group 2: Company Confidence and Product Pipeline - The company remains confident in the benefit-risk profile of aficamten and anticipates a differentiated label and risk mitigation profile upon potential FDA approval [3]. - Cytokinetics is advancing a pipeline of potential new medicines for cardiac muscle dysfunction, including aficamten, omecamtiv mecarbil, CK-586, and CK-089 [3].