Israeli biopharm SpliSense has announced that both the FDA and the EMA have granted orphan drug designation to the company’s SPL84-23-1 inhaled antisense oligonucleotide (ASO) for the treatment of cystic fibrosis associated with the 3849+10 kb C-to-T mutation. According to the company, preclinical studies have demonstrated that treatment with SPL84-23-1 can “enable production of a fully functional CFTR protein and therefore has the potential to treat the CF disease associated with the 3849 mutation.”
The company’s web site says, “SpliSense treatment is delivered non-invasively, by inhalation, carrying large doses to the lungs with minimal systemic exposure. Our treatment’s efficacy is backed by research showing that inhaled ASOs distribute effectively with proper cellular penetration, minimal systemic absorption and no toxicity in mice, non-human primates (NHPs) and humans. The treatment is administered weekly, reducing patients’ treatment burden.”
SpliSense CEO Gili Hart commented, “The orphan designations we received for our lead drug candidate both from the FDA and EMA are important milestones toward addressing the urgent, unmet needs of patients living with cystic fibrosis. We are looking forward to initiating the clinical trial with SPL84-23, that is expected to commence in mid-2022 in multiple centers in the US, Europe and Israel.”
Read the SpliSense press release.
