EC Grants Orphan Drug Designation to Vico Therapeutics’ Oligonucleotide SCA Therapy

The European Commission (EC) has granted Vico Therapeutics with orphan drug designation for its investigational antisense oligonucleotide therapy (VO659) for the treatment of Spinocerebellar Ataxia (SCA).

According to a Feb 10, 2021 press release, the orphan designation was granted based on a positive opinion from the European Medicines Agency Committee for Orphan Medicinal Products. A previous orphan designation has also been granted for VO659 for the treatment of Huntington Disease.

“[SCA] 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, chief medical officer at Vico, in the press release.“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.”