The company generates revenue through royalties from drugs like SPINRAZA and sales of TEGSEDI and WAYLIVRA. Its innovative approach uses modified RNA to address genetic causes of diseases ...

European regulators have recommended a licence for Akcea and Ionis’ ultra-rare disease drug Waylivra, a therapy that the FDA rejected for the US market last year. Waylivra (volanesorsen ...

NICE assessed Waylivra using its highly specialised technology pathway, which in most cases allows for a cost-effectiveness threshold of £100,000 per Quality Adjusted Life Year.