Ninerafaxstat is a first-in-class therapy targeting cardiac energetics and is currently being evaluated in the Phase 2b FORTITUDE-HCM trial in patients with symptomatic nHCM

Orphan Drug Designation provides regulatory and development incentives, including enhanced FDA interaction, potential financial advantages, and seven years of market exclusivity upon approval

Imbria Pharmaceuticals, Inc. (Imbria), a clinical-stage company developing innovative therapeutics for cardiovascular diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ninerafaxstat for the treatment of symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM).

Ninerafaxstat, Imbria’s first-in-class investigational oral therapy designed to improve the heart’s ability to produce energy efficiently, is currently being evaluated in the Phase 2b FORTITUDE-HCM clinical trial (NCT07023614) in patients with symptomatic nHCM.

“Receiving Orphan Drug Designation for ninerafaxstat is an important milestone as we advance its development for patients living with symptomatic nHCM, a population with significant unmet medical need,” said Alvin Shih, MD, Chief Executive Officer of Imbria. “Despite recent progress in HCM, there are still no approved therapies for nHCM, and patients continue to experience debilitating symptoms, including shortness of breath, fatigue, and exercise intolerance. By improving cardiac energetics, ninerafaxstat represents a differentiated approach to addressing nHCM, with the potential to be used as a standalone therapy or in combination with other treatments. We look forward to advancing ninerafaxstat through the ongoing FORTITUDE-HCM trial and anticipate reporting topline data in the first half of 2027.”

The FDA grants Orphan Drug Designation to therapies that are being developed to treat rare diseases and conditions that affect fewer than 200,000 people in the U.S. The designation provides certain incentives, such as assistance in the drug development process, tax credits toward the cost of clinical trials, and prescription drug user fee waivers. Upon approval, products with Orphan Drug Designation may be eligible for seven years of market exclusivity for the designated indication, independent of patent protection. This exclusivity complements Imbria’s existing intellectual property portfolio for ninerafaxstat, which includes multiple granted patents extending to 2038 and beyond.