Ionis Pharmaceuticals Nabs Orphan Drug Designation for Huntington’s Candidate

The FDA has blessed Ionis Pharmaceuticals’ Huntington’s disease candidate with an orphan drug designation.

IONIS-HTTRx would be the first treatment to combat the root cause of the fatal neurological disease. It works by cutting the amount of huntingtin protein, the protein that overproduces in Huntington’s disease patients, harming brain neurons.

Also deemed an orphan drug in Europe, the rare-disease designation grants Ionis access to seven years of market exclusivity for the therapy if approved, tax credits and FDA guidance on medical research.