Ionis Gets FDA Orphan Drug Status For IONIS-HTT Rx In Huntington's Disease

(RTTNews) - Ionis Pharmaceuticals, Inc. (IONS) said the U.S. Food and Drug Administration has granted Orphan Drug Designation to IONIS-HTTRx for the treatment of patients with Huntington's disease or HD.

IONIS-HTTRx is the first therapy to enter clinical development designed to directly target the cause of the disease, by reducing the production of the protein responsible for HD. It has also been granted orphan drug designation by the European Medicines Agency for the treatment of patients with HD.

The Orphan Drug Act provides for economic incentives to encourage the development of drugs for diseases affecting fewer than 200,000 people in the US. Orphan drug designation entitles seven years of market exclusivity in the U.S. if market approval is granted for IONIS-HTTRx for the treatment of patients with HD.

Additional incentives include tax credits related to clinical trial expenses, an exemption from the FDA-user fee, and FDA assistance in clinical trial design.

Roche and Ionis are collaborating to develop antisense drugs to treat HD.