FDA grants Huntingdon disease treatment orphan-drug designation

pharmafile | July 30, 2021 | News story | Medical Communications  

The FDA has granted VICO’s investigational therapy for Huntingdon disease, VO659, orphan-drug designation.

This news comes just one month after VICO announced that VO659 had received orphan-drug designation for the treatment of spinocerebellar ataxia (SCA).

VO659 is VICO’s investigational antisense oligonucleotide (AON) therapy for the treatment of Huntington’s disease, a rare inherited neurodegenerative disorder which currently has no effective treatments.

Rupert Sandbrink, Chief Medical Officer at VICO, said: “Huntington’s disease, like many types of SCA, belongs to the group of polyglutamine disorders which are rare genetic and progressive brain diseases.

“Patients affected by Huntington’s disease experience motor disturbances, personality changes, and dementia, leading to increasing disability, loss of independence, and reduced survival. Only very limited, symptomatic treatment options are currently available for patients with this devastating disease.

“Our investigational RNA modulating therapy is aimed to be a disease modifying treatment for polyglutamine disorders, designed to lower the mutant polyglutamine protein levels that cause these neurodegenerative diseases.”

 “We are delighted that FDA has granted this orphan-drug designation, following the ODD for SCA one month ago. This is affirmation of the potential of our AON approach.”

The FDA’s Office of Orphan Products Development grants Orphan Drug Designation to drugs and biologics that are defined as those intended for the safe and effective treatment, diagnosis, or prevention of rare diseases/disorders that affect fewer than 200,000 people in the US.

The designation allows VICO to qualify for a number of incentives, including seven years of market exclusivity upon regulatory approval; exemption from FDA application fees for Huntington’s Disease; and tax credits for qualified clinical trials.

Lilly Subbotin

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