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SpliSense Granted FDA Fast Track Designation for SPL84 in Cystic Fibrosis Therapy

NextEdge Admin

31 May 2024

3 min 6 sec

SpliSense, headquartered in Jerusalem, has garnered Fast Track Designation from the U.S. Food and Drug Administration (FDA) for its lead antisense oligonucleotide (ASO) product, SPL84, aimed at treating cystic fibrosis (CF) patients carrying the 3849+10 kilobase (Kb) C->T splicing mutation in the CFTR gene. This designation underscores the urgent need for effective treatments in CF, a condition with limited therapeutic options.

Gili Hart, PhD, CEO of SpliSense, emphasized the significance of this milestone, highlighting the severity of CF and the inadequacy of current treatments for patients with the specified mutation. The FDA’s recognition expedites the development process, potentially accelerating access to life-changing therapies.

Fast Track Designation streamlines the regulatory pathway for drugs addressing critical medical needs. It fosters closer collaboration between the FDA and drug developers, expediting the review process and facilitating communication to hasten the availability of vital treatments.

CF is a genetic disorder characterized by mutations in the CFTR gene, affecting chloride channel production in the lungs and other tissues. While recent years have witnessed advancements in CF care with the introduction of CFTR modulators, these treatments do not benefit all patients, especially those with specific mutations like 3849+10 Kb C->T.

SPL84, SpliSense’s innovative ASO therapy, targets mutated CFTR RNA, aiming to restore normal protein function. Administered via inhalation, SPL84 is designed to reach lung cells directly, potentially enabling the production of functional CFTR proteins. Promising preclinical results have demonstrated SPL84’s ability to fully restore CFTR activity in pharmacological models.

SpliSense’s platform extends beyond CF to address a range of pulmonary diseases, including muco-obstructive diseases and idiopathic pulmonary fibrosis (IPF). By leveraging ASOs, the company aims to correct genetic mutations or deficiencies, targeting the underlying causes of disease.

With SPL84 advancing through a global Phase 2 study, SpliSense is poised to revolutionize CF treatment and expand its innovative RNA-based therapies to address significant unmet needs in pulmonary medicine.