Orphan Drug Status Granted to Huntington's First Target-Directed Tx
Ionis Pharmaceuticals announced that the Food and Drug Administration has granted Orphan Drug designation to IONIS-HTT for the treatment of Huntington's disease (HD).
IONIS-HTT is the first target-directed therapy in clinical development designed to treat the underlying cause of Huntington's disease, a rare, genetic neurological disorder.
IONIS-HTT, a Gen. 2.0+ antisense drug, directly targets the cause of HD by reducing the production of all forms of the huntingtin (HTT) protein. This mechanism offers a unique approach for the treatment of the disease.
For more information visit Ionispharma.com.