EU committee approves Akcea-Ionis drug for rare genetic disease


March 1 (Reuters) – An advisory committee of the European Medicines Agency (EMA) on Friday recommended conditionally approving a drug from Akcea Therapeutics Inc and Ionis Pharmaceuticals Inc that aims to treat a rare genetic disease that causes fat accumulation in the blood.

The nod from EMA’s Committee for Medicinal Products for Human Use is a shot in the arm for the companies after their shares plunged in August when the U.S. Food and Drug Administration declined to approve the drug, Waylivra.

Waylivra is designed to treat familial chylomicronemia syndrome, a rare genetic disorder that can cause a potential fatal inflammation of the pancreas.

Regulators have in the past raised safety concerns such as low platelet count associated with the drug, but there is a lack of available treatments for the condition.


(Reporting by Pushkala Aripaka in Bengaluru; Editing by Shounak Dasgupta)


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