FDA grants Fast Track designation to SpliSense’s SPL84 inhaled antisense oligonucleotide for CF

According to SpliSense, the FDA has granted Fast Track designation to the company’s SPL84 inhaled antisense oligonucleotide for the treatment of cystic fibrosis. SPL84 had already received Orphan Drug designation from both the FDA and the EMA. Earlier this year, the FDA cleared an IND for a Phase 2 trial of SPL84 in patients with cystic fibrosis associated with the 3849+10 kb C-to-T mutation.

SpliSense CEO Gili Hart commented, “Fast Track designation for SPL84 is an important acknowledgement by the FDA of the critical need to find an effective treatment for CF patients carrying the 3849+10 kilobase (Kb) C->T mutation, a serious and life-threatening condition with very poor treatment options. The recent IND clearance we received from the FDA together with this Fast Track designation for SPL84, currently being evaluated in a global Phase 2 study, will allow us to expedite the development of a potentially life-changing treatment for people with CF carrying the 3849+10 Kb C->T mutation, and expand our unique technology to additional pulmonary indication where there is a significant unmet need.”

Read the SpliSense press release.

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