Porphyria

News

Disc maps out US filing for porphyria drug bitopertin

Disc Medicine has charted a route to market for bitopertin, its drug candidate for rare disease erythropoietic protoporphyria (EPP), after meeting with the FDA.

News

Alnylam gets NICE backing for porphyria therapy Givlaari

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

News

FDA approves Alnylam's second RNAi drug - with $442K annual ...

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.

News

Alnylam to file gene-silencing porphyria drug

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

Disc maps out US filing for porphyria drug bitopertin

Alnylam gets NICE backing for porphyria therapy Givlaari

FDA approves Alnylam's second RNAi drug - with $442K annual ...

Alnylam to file gene-silencing porphyria drug

Neurogene drops high-dose arm in Rett study after death

Coming soon: The Life Sciences Industry Report 2025 – insigh...

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Porphyria

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