US FDA Award VICO with Therapeutics Orphan-Drug Designation for VO659

VICO Therapeutics has announced that the US Food and Drug Administration's Office of Orphan Products Development (OOPD) had granted the company a patent.

FREMONT, CA: VICO Therapeutics, a biotech firm based in Leiden, the Netherlands, focuses on developing RNA modulating therapies for rare neurological disorders, announces that the US Food and Drug Administration's Office of Orphan Products Development (OOPD) had granted the company a patent. The Food and Medication Administration (FDA) has designated VO659, VICO's investigational antisense oligonucleotide (AON) therapy for the treatment of spinocerebellar ataxia (SCA), as an orphan drug. The European Commission granted VICO orphan drug classification for VO659 in SCA in February.

Rupert Sandbrink, MD PhD., Chief Medical Officer at VICO, stated: "Spinocerebellar ataxia belongs to the group of polyglutamine disorders which are debilitating and progressive diseases, leading to significant impairment of mobility and multiple other daily activities of patients suffering from these conditions. Currently, no disease modifying treatments are available for these patients. Our investigational RNA modulating therapy is designed to lower the mutant protein levels causing these neurodegenerative diseases."

"We're excited to obtain orphan-drug designation from also the FDA, following the EC grant earlier this year. This deeply underserved patient population deserves new therapies, and we look forward to advancing VO659, our therapy that holds great potential for addressing their unmet needs. It also recognizes the potential of our AON approach. This is yet another important step forward as we prepare for our first in human trials, expected to start in 2022."

Orphan Drug Designation is granted by the FDA's Office of Orphan Products Development to medicines and biologics designed for the safe and efficient treatment, diagnosis, or prevention of rare diseases/disorders that impact fewer than 200,000 persons in the United States. VICO will be eligible for various benefits due to the designation, including seven years of market exclusivity upon regulatory clearance, a waiver of FDA application fees for spinocerebellar ataxia, and tax credits for approved clinical trials.