FDA Grants Orphan Drug Designation to VICO Therapeutics for Huntington’s Disease Drug

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VICO Therapeutics’ drug candidate, VO659, for treating Huntington’s Disease was granted orphan drug designation by FDA.

On July 29, 2021, VICO Therapeutics, a Netherlands-based biotech company focused on the development of RNA-modulating therapies for rare neurological disorders, announced that FDA had granted orphan drug designation (ODD) for VO659, an investigational antisense oligonucleotide therapy (AOT), in the treatment of Huntington’s disease (HD).

HD is a rare degenerative brain disease that results in motor disorders, impaired cognition, dementia, and various psychiatric manifestations. It is caused by a mutation to the CAG-trinucleotide repeat in the coding region of exon 1 of the HTT gene, resulting in a mutant huntingtin protein with an elongated polyglutamine (polyQ) stretch at its N-terminus. This grants a toxic gain-of-function to mutant protein forms, which results in widespread neuronal death.

VO659 is designed to suppress mutant proteins with the intent of slowing or halting the progression of disease. A month prior to this announcement, the drug had also received orphan drug designation for the treatment of spinocerebellar ataxia, a group of hereditary polyQ disorders that result in similar neuron degeneration.

ODD is granted to drugs intended for the safe and effective treatment, diagnosis, or treatment of rare (fewer than 200,000 cases) diseases or disorders. This designation grants various benefits, including seven years of market exclusivity following regulatory approval, exemption from FDA application fees for Huntington’s disease, and tax credits for qualified clinical trials.


“We are delighted that [FDA] has granted this orphan drug designation following the ODD for SCA one month ago,” said Rupert Sandbrink, chief medical officer at VICO, in a company press release. “This is affirmation of the potential of our [antisense oligonucleotide platform] approach.”

Source: VICO Therapeutics