The Israeli biopharmaceutical company SpliSense’s cystic fibrosis treatment has been granted “orphan drug” designation by the U.S. Food and Drug Administration.
Orphan drug designation is applied by the FDA and EMA to medicine and biologic technology intended to treat rare conditions.“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,” SpliSense CEO Gili Hart said in a press release.
“We are looking forward to initiating the clinical study in the patient population in the US, Europe and Israel, which is aimed to be initiated later this year.”
The product in question is an ASO (antisense oligonucleotide) called SPL84-23, a short and precisely targeted RNA strand that corrects a specific mutation that causes cystic fibrosis: the 3849 mutation in the CFTR gene. A dosage of these ASOs are inhaled into the lungs, where they are incorporated into the cells there and begin to produce corrected mRNA strands and normal proteins, potentially leading to significant improvement in lung function.