HAYA Therapeutics Receives FDA Fast Track Designation for HTX-001 in Non-Obstructive Hypertrophic Cardiomyopathy
Wednesday, July 29, 2026
HAYA Therapeutics has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for HTX-001, its investigational therapy for the treatment of symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM).
HTX-001 is a first-in-class antisense oligonucleotide designed to reduce the expression of WISPER, a long non-coding RNA that is overexpressed in hypertrophic cardiomyopathy and linked to cardiac stress. The therapy is intended to target cardiac myofibroblasts and reprogramme these cells from a disease-associated state towards a healthier phenotype, with the aim of addressing the underlying fibrosis that contributes to the condition.
The investigational treatment is currently being evaluated in a Phase 1a/b clinical trial, with the first patient cohort having received treatment in May.
The FDA's Fast Track designation is intended to support the development and review of medicines for serious conditions where there is an unmet medical need. The designation enables closer interaction with the FDA during clinical development and may provide eligibility for accelerated approval, priority review and rolling review if the relevant criteria are met.
Non-obstructive hypertrophic cardiomyopathy accounts for an estimated 30% to 60% of all hypertrophic cardiomyopathy cases. Current treatment options mainly focus on symptom management and do not directly target the fibrotic changes or diastolic dysfunction that drive disease progression.
HTX-001 remains an investigational therapy and has not been approved by the U.S. FDA, the European Medicines Agency or any other regulatory authority. Its safety and clinical effectiveness are still being evaluated in ongoing clinical studies.
Source: businesswire.com
