Ionis Pharmaceuticals Receives Orphan Drug Designation for HTTRx

January 5, 2016
BioPharm International Editors

Ionis Pharmaceuticals receives orphan drug designation for HTTRx for the treatment of Huntington’s disease.

FDA has granted Ionis Pharmaceutical’s orphan drug designation for IONIS-HTTRx, for the treatment of patients with Huntington’s disease (HD). According to a Jan. 5, 2016 press announcement from the company, IONIS-HTTRx is the first therapy to enter clinical development that is designed to directly target the cause of the disease by reducing the production of the protein responsible for HD.

IONIS-HTTRx is a Gen. 2.0+ antisense drug in development for the treatment of Huntington's disease. The drug is designed to reduce the production of all forms of the HTT protein, which is the protein responsible for HD. HD is referred to as a triplet repeat disorder and is one of a large family of genetic diseases in which certain gene sequences are mistakenly repeated. In HD, the gene that encodes for the HTT protein contains a trinucleotide sequence that is repeated in the gene more than 36 times. The resulting HTT protein is toxic and gradually damages neurons in the brain

"HD is a rare genetic neurological disease in which patients experience deterioration of both mental abilities and physical control. Although the toxic protein produced from the huntingtin (HTT) gene in HD patients has been a target of interest for many years, IONIS-HTTRx is the first therapy to enter clinical development that is designed to treat the underlying cause of this fatal disease,” said C. Frank Bennett, PhD, senior vice-president of research at Ionis Pharmaceuticals. “The granting of Orphan Drug Designation in both the US and Europe highlights the significant need for a drug that could transform the treatment of HD."

The drug has also been granted orphan drug designation by the European Medicines Agency for the treatment of patients with HD. 

Source: Ionis Pharmaceuticals