Shares in Catalyst Pharmaceuticals have dived after the US regulator approved a potential competitor to its rare disease drug Firdapse, which has been at the centre of a drug pricing row.
Overview
Launching orphan medicines excellently will become even more important over the next five years as more patients with rare diseases have pharmacotherapies available.
UK biotech Mereo BioPharma is hoping the coming months will transform its fortunes as it prepares for potentially pivotal data for a brittle bone disease drug.
NICE has recommended that Akcea’s hereditary transthyretin-related amyloidosis (hATTR) treatment Tegsedi be made available on the NHS in England, ahead of a rival therapy from Alnylam.
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
A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.
