FDA grants Orphan Drug Designation to IONIS-HTTRx

Posted: 5 January 2016 | | 4 comments

IONIS-HTTRx is a Gen. 2.0+ antisense drug in development for the treatment of Huntington’s disease…

The US Food and Drug Administration has granted Orphan Drug Designation for IONIS-HTTRx for the treatment of Huntington’s disease.

huntington's disease

IONIS-HTTRx is a Gen. 2.0+ antisense drug. It 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. In Huntington’s disease, 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.

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

IONIS-HTTRx is the first therapy designed to treat the underlying cause of the disease

“Huntington’s disease 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 Huntington’s disease 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. 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,” said C. Frank Bennett, Ph.D., senior vice president of research at Ionis Pharmaceuticals.

Orphan drug designation entitles seven years of market exclusivity in the United States if market approval is granted for IONIS-HTTRx for the treatment of patients with Huntington’s disease. Additional incentives include tax credits related to clinical trial expenses, an exemption from the FDA-user fee, and FDA assistance in clinical trial design.

Ionis Pharmaceuticals and Roche are collaborating on the development of the treatment. To date, Ionis has earned $52 million in upfront and milestone payments from its relationship with Roche and is eligible to earn additional milestone payments as the drug progresses in development, as well as royalties on sales of IONIS-HTTRx if it is commercialised. Roche has the option to license IONIS-HTTRx from Ionis through the completion of the Phase 1/2a study.

4 responses to “FDA grants Orphan Drug Designation to IONIS-HTTRx”

  1. chris dagner says:

    as far back as my memory takes me this disease has ravaged my family. Grandfather, Mother, 3 siblings have passed. At present I have a Niece and Nephew, children of my deceased youngest Sister that have tested positive for the HD Gene. I certainly would be interested in any trials taking place in the U.S. Both of the children are in their mid-twenties which seems to follow the onset in my family historically, Any information would, of course, be greatly appreciated…thank you

  2. Jerry Klarr says:

    Sounds very promising. I have a wife and brother in law that have the disease. Are there trials yet being set up in the US?

  3. Joe Terrell says:

    Hoping this proves beneficial. Friends dealing with Huntington’s

  4. carole Sue Fine says:

    Please keep me informed. My husband had late on sight of HHD (70). And passed away this summer at 84.
    In his later years his health deteriorated dramatically. At the end here was “that other guy ” vary loopy and not his usual nice self.

    Please keep me on your list.
    Thank you,
    Sue Fine
    Sfine [email protected] .net

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