BRIEF—Ionis and Roche's Huntington's therapy granted PRIME designation

3 August 2018

Ionis Pharmaceuticals announced on Thursday that its novel Huntington's therapy, licensed to Roche, had been granted PRIME designation by the European Medicines Association (EMA).

The drug, which is the first to target the mutant huntingtin gene that is the root cause of the disease, will be entitled to an accelerated priority review by regulators as a result.

"We are very pleased that the EMA has granted PRIME designation for RG6042, as there is an urgent medical need to find treatment options for families affected by Huntington's disease," said Sandra Horning, chief medical officer at Roche."

"Preliminary data on RG6042 were the first to show that levels of toxic mutant huntingtin protein can be lowered in adults with Huntington's disease, and we are working closely with the EMA and other health authorities to initiate a global phase III study as soon as possible."



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