According to SpliSense, the European Medicines Agency has granted early Priority Medicines designation to the company’s SPL84 inhaled antisense oligonucleotide for the treatment of cystic fibrosis in patients with a 3849+10Kb C>T mutation in the CFTR gene. SPL84, a weekly therapy delivered via nebulization, has previously received orphan drug designation from both the EMA and the FDA and Fast Track designation from the FDA for this indication.
In September 2025, the company announced data from its Phase 2a SPL84-002 trial in CF patients with the mutation, and the current announcement says that the company will advance the inhalation solution to Phase 2b study. In addition to SPL84, SpliSense’s pipeline includes SPL5AC for muco-obstructive diseases including COPD, asthma, and non-CF bronchiectasis and SPL5B for idiopathic pulmonary fibrosis, which it plans to advance into the clinic this year.
SpliSense CEO Gili Hart commented, “This PRIME designation represents a major regulatory milestone for SpliSense and for the SPL84 program. It reflects the strength of our mechanistic, non-clinical, and emerging clinical data, and reinforces the potential of SPL84 to deliver meaningful benefit to people with cystic fibrosis who continue to represent a significant unmet medical need.”
Read the SpliSense press release
