UK cost-effectiveness watchdog NICE is set to recommend NHS use of Alnylam's gene-silencing therapy Givlaari in England and Wales for the rare disease acute hepatic porphyria (AHP), after i
Alnylam has had a second RNA interference (RNAi) drug approved by the FDA, to treat the ultra-rare disease acute hepatic porphyria, with an ultra-high price tag to match.
Alnylam is to file its givosiran with regulators to treat a form of the blood disease porphyria, applying its gene silencing technology to the condition for the first time, following suppor
The EMA's human medicines committee has recommended against approval of Eisai and Biogen's Alzheimer's disease therapy Leqembi, after regulators in the US, China and Japan gave a green ligh