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- Amgen submits new analysis to FDA to defend continued availability of TavneosAmgen has provided the FDA with a fresh analysis concerning its rare-disease therapy Tavneos, aiming to address the agency’s concerns. The company says the independent reevaluation supports the dr…
- 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…