Rare Diseases

GSK jilts Ionis rare disease drug as FDA filing nears

Emma Walmsley
GSK CEO Emma Walmsley

GlaxoSmithKline has jilted Ionis Pharmaceuticals just before the pair was due to file for approval of inotersen. The action furthers recently installed GSK CEO Emma Walmsley’s plan to purge the Big Pharma of its rare disease programs.

Ionis recently brought the transthyretin-targeting antisense drug through a phase 3 program that teed it up to file for approval in polyneuropathy due to hereditary TTR amyloidosis this year. But it will now have to take the drug the final mile unpartnered after GSK declined its option to pick up it and another asset, IONIS-FB-LRx.

Management at Ionis put a positive spin on the news.

“We are pleased to move forward these two important drugs ourselves. We are prepared to independently advance inotersen and remain on track to file for marketing approval…