Release Date: 11-Sep-2021
US FDA has granted Orphan Drug Designation for VO659 for the treatment of patients with Huntington’s disease (HD). VO659 is developed to suppress mutant proteins with the intent of reducing or halting the progression of disease. Previously, the drug had also received orphan drug designation for the treatment of spinocerebellar ataxia, a group of hereditary polyQ disorders which results in similar neuron degeneration.
The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S and it also gives several benefits, like tax credits for qualified clinical trials, potential seven years of market exclusivity after approval and exemption from user fees.
HD is a rare degenerative brain disease that results in impaired cognition, dementia, motor disorders, and various psychiatric manifestations. HD 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.