Pharma Focus America

SpliSense's SPL84 Receives FDA Fast Track Designation for Cystic Fibrosis Therapy

Thursday, May 30, 2024

SpliSense, a biotechnology company in the clinical stage, is focused on developing RNA-based therapies for pulmonary diseases like cystic fibrosis (CF), obstructive pulmonary diseases such as COPD and asthma, as well as idiopathic pulmonary fibrosis (IPF). Today, the company announced a significant development: the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to SPL84 for CF.

SPL84 is the company's primary antisense oligonucleotide (ASO) product designed to address CF in patients carrying the 3849+10 kilobase (Kb) C->T splicing mutation within the transmembrane conductance regulator (CFTR) gene.

SpliSense, emphasized the importance of this designation, stating, "Fast Track Designation for SPL84 is an important acknowledgment 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." She further highlighted that the recent FDA IND clearance, coupled with the Fast Track designation for SPL84, currently undergoing evaluation in a global Phase 2 study, will accelerate the development of a potentially transformative treatment for CF patients with the 3849+10 Kb C->T mutation, while also paving the way for addressing other pulmonary indications with significant unmet needs.

Fast Track Designation is a regulatory status granted by the FDA to expedite the development and review of drugs meant to treat serious conditions with unmet medical needs. It facilitates enhanced communication and collaboration between the FDA and drug developers, potentially hastening the availability of life-saving treatments to patients.

SPL84 operates through short, precisely targeted RNA stretches known as Antisense Oligonucleotides (ASOs) to modulate specific mRNA sequences or rectify various mutations within the target mRNA. Administered directly to the lungs via inhalation, SPL84 binds specifically to the mutated CFTR RNA, thereby modulating the targeted sequence and potentially enabling the production of fully functional CFTR proteins. This unique delivery mechanism is designed to drive the production of corrected CFTR mRNA and ultimately fully functional CFTR proteins within lung cells. Notably, SPL84 has demonstrated the ability to fully restore CFTR activity in the CF gold standard pharmacological model. Currently, SPL84 is undergoing evaluation in a global Phase 2 study for the treatment of CF patients carrying the 3849+10 kilobase (Kb) C->T mutation through a weekly inhalation regimen.



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