FDA Grants Orphan Drug Designation For VO659

Release Date: 18-Sep-2021

US FDA has designated Orphan Drug Designation VO659, an investigational antisense oligonucleotide gene-silencing therapy for Huntington’s disease. VO659 such as, Antisense oligonucleotides are artificially created nucleotides that bind to the HTT messenger RNA and prevent it from being translated into a protein. RNA is a molecule which serves as a template for cells to make the corresponding protein.


Through in this mechanism, the gene is “silenced” and the abnormally long huntingtin proteins are no longer produced. Huntington’s disease is caused by mutations in the huntingtin (HTT) gene, which expand the gene by introducing long segments of repeating CAG nucleotides.


Earlier, VO659 received orphan drug designation for the treatment of spinocerebellar ataxia (SCA) by the FDA, and for the treatment of Huntington’s disease and SCA from the European Commission, based on positive evaluations from the European Medicines Agency.


The FDA’s Orphan Drug Program provides financial incentives to companies developing drugs for rare diseases and conditions. Through the program, eligible drugs can be granted tax credits and other benefits.

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