Vico Therapeutics, a Leiden, the Netherlands, based biotech company focusing on the development of RNA modulating therapies for rare neurological disorders, today announced that the European Commission (EC) has granted orphan drug designation for VO659, Vico’s investigational antisense oligonucleotide therapy for the treatment of Spinocerebellar Ataxia (SCA). The orphan designation was based on a positive opinion from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP). Vico previously received orphan drug designation for VO659 in Huntington Disease.
“Spinocerebellar Ataxias make up a great part of the so called polyglutamine disorders, debilitating and progressive diseases, leading to significant impairment of mobility, speech, and multiple other daily activities of patients suffering from this condition. So far, there is no treatment which can halt the progression of these diseases,” said Rupert Sandbrink M.D. Ph.D., Chief Medical Officer at Vico.
“Our investigational RNA modulating therapy is designed to lower the mutant protein levels causing these neurodegenerative diseases. We’re pleased to receive orphan drug designation, which recognizes both the unmet medical need for patients living with spinocerebellar ataxias, and the potential of our approach.”
Qualified for orphan drug designation are drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that impact fewer than 5 in 10,000 patients in the European Union. Orphan drug designation gives developing companies certain benefits, including clinical protocol assistance, reduced regulatory fees, research grants and 10 years of market exclusivity following regulatory approval.