FDA Grants HAYA’s nHCM Therapy Fast Track Designation

Rare Daily Staff

The U.S. Food and Drug Administration granted HAYA Therapeutics Fast Track designation for its lead RNA-targeting program, HTX-001, for the treatment of symptomatic nonobstructive hypertrophic cardiomyopathy.

The investigational therapy, a first-in-class antisense oligonucleotide designed to target long noncoding RNA (lncRNA), is currently being evaluated in a Phase 1a/b clinical trial, with initial patient dosing completed in May.

Nonobstructive hypertrophic cardiomyopathy (nHCM) is a form of hypertrophic cardiomyopathy in which the heart muscle is thickened but does not cause significant obstruction of blood flow out of the heart. nHCM accounts for an estimated 30 percent to 60 percent of all hypertrophic cardiomyopathy cases and is characterized by left ventricular hypertrophy, impaired diastolic function, and significant fibrosis. Despite advances in understanding the disease, currently available therapies do not directly target the fibrotic remodeling that drives disease progression.

HTX-001 works by downregulating WISPER, a cardiac stress–associated lncRNA implicated in fibrosis and disease progression in hypertrophic cardiomyopathy. By targeting WISPER in cardiac myofibroblasts, the therapy aims to reprogram these cells away from a pathological, fibrotic state toward a healthier phenotype—an approach that could differentiate it from existing treatments that primarily address symptoms rather than underlying disease biology.

Fast Track designation is intended to accelerate the development and review of therapies for serious conditions with unmet medical need, enabling more frequent FDA interactions and potential eligibility for accelerated approval, priority review, and rolling submissions.

“This regulatory designation recognizes the potential of HTX-001 to offer a differentiated therapeutic approach for patients with nHCM,” said Samir Ounzain, CEO and co-founder of HAYA Therapeutics.

Photo: Samir Ounzain, CEO and co-founder of HAYA Therapeutics