Rare Disease Report

FDA Grants Orphan Drug Designation to Huntington Disease Treatment

JANUARY 06, 2016
RDR Staff
The FDA granted orphan drug designations to IONIS-HTT, Ionis Pharmaceuticals’ treatment of patients with Huntington's disease (HD). Ionis Pharmaceuticals was formerly known as Isis Pharmaceuticals.
Huntington's Disease is an inherited brain disorder that results in the progressive loss of both mental and motor control. Symptoms usually appear between the ages of 30 to 50, and worsen over a 10 to 25 year period until the patient eventually dies. In Huntington’s disease, the gene that encodes for the huntingtin protein contains a trinucleotide sequence that is repeated in the gene multiple times resulting in the huntingtin protein being toxic to neurons. IONIS-HTT is a Gen. 2.0+ antisense drug designed to reduce the production of all forms of the huntingtin protein.
In April 2013, Ionis and Roche entered into an alliance to develop treatments for Huntingtons based on the antisense technology and utilizing Roche's scientific expertise in developing neurodegenerative therapeutics. Under the agreement, Roche has the option to license IONIS-HTT from us through the completion of the Phase 1/2a study.
IONIS-HTT has also been granted orphan drug designation by the European Medicines Agency for the treatment of patients with Huntington’s disease.
"HD is a rare genetic neurological disease in which patients experience deterioration of both mental abilities and physical control,” said C Frank Bennett, PhD, senior vice president of research at Ionis Pharmaceuticals. “Although the toxic protein produced from the Huntington (HTT) gene in HD patients has been a target of interest for many years, IONIS-HTT is the first therapy to enter clinical development that is designed to treat the underlying cause of this fatal disease. 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 Huntington’s disease."


Ionis Pharmaceuticals Receives Orphan Drug Designation from the US FDA for IONIS-HTT Rx for the Treatment of Patients with Huntington's Disease [news release]. Carlsbad, CA; Ionis Pharmaceuticals: January 5, 2015. http://www.prnewswire.com/news-releases/ionis-pharmaceuticals-receives-orphan-drug-designation-from-the-us-fda-for-ionis-htt-rx-for-the-treatment-of-patients-with-huntingtons-disease-300198992.html

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