source
- Amgen seeks FDA hearing to keep rare kidney drug Tavneos amid safety concernsAmgen has asked the U.S. Food and Drug Administration for a hearing to argue against a proposed withdrawal of its rare‑disease drug Tavneos. The company submitted a data package containing real‑wo…
- EU regulators back Minoryx’s ALD therapy while rejecting Zevra’s rare-disease drugThe EU Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion on Minoryx Therapeutics' drug for adrenoleukodystrophy, marking a significant regulatory step after previous c…