Halia Therapeutics’ Ofirnoflast Gains FDA Orphan Drug Designation

Halia Therapeutics, Inc., a clinical-stage biopharmaceutical company based in Lehi, Utah, announced on October 23, 2025, that its investigational drug, ofirnoflast (HT-6184), has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA). This designation is significant for the treatment of Myelodysplastic Syndromes (MDS), a group of bone marrow disorders that lead to ineffective blood cell production and pose an increased risk of progression to acute myeloid leukemia (AML).

The Orphan Drug Designation is granted by the FDA to encourage the development of treatments for rare diseases affecting fewer than 200,000 individuals in the United States. “This designation underscores the potential of our approach in Myelodysplastic Syndromes and supports our commitment to developing new treatment options for patients living with MDS,” stated David Bearss, PhD, Chief Executive Officer of Halia Therapeutics.

Ofirnoflast represents a novel treatment strategy, acting as a selective NEK7 allosteric modulator. It is designed to prevent the formation and promote the disassembly of the NLRP3 inflammasome, a significant contributor to chronic inflammation in various diseases. In the context of MDS, the activation of the inflammasome is increasingly recognized as a critical factor in ineffective hematopoiesis and bone marrow failure.

By targeting NEK7, ofirnoflast aims to restore immune balance and enhance blood cell production without the broad immunosuppressive effects typical of many current treatments. “Inflammasome biology represents a promising frontier for hematologic innovation,” remarked Alan F. List, MD, a member of Halia Therapeutics’ Scientific Advisory Board and former President and CEO of Moffitt Cancer Center. He emphasized that ofirnoflast’s unique approach focuses on modulating the inflammatory drivers of MDS rather than merely addressing its symptoms.

The FDA’s Orphan Drug Act provides several benefits for therapies awarded this status, including tax credits for clinical trials, exemption from certain FDA user fees, and the potential for seven years of U.S. market exclusivity upon approval. Additionally, the FDA offers grant programs to support clinical research aimed at advancing therapies for rare diseases.

Understanding Myelodysplastic Syndromes

Myelodysplastic Syndromes are characterized by defective blood cell formation, resulting in complications such as anemia, increased infection risk, and bleeding disorders. Primarily affecting older adults, MDS can progress to acute myeloid leukemia (AML). Current treatments, including hypomethylating agents and growth factors, often yield limited results and do not adequately address the underlying inflammatory processes of the disease.

Future Prospects for Ofirnoflast

As the lead investigational compound from Halia Therapeutics, ofirnoflast is positioned as a first-in-class NEK7 modulator. The drug is currently being evaluated across multiple disease areas, including hematologic disorders. Halia Therapeutics aims to leverage genetic resilience to restore the body’s natural ability to resolve inflammation by targeting the NEK7–NLRP3 inflammasome axis. This strategy has the potential to address a spectrum of chronic inflammatory and degenerative diseases.

For more information about Halia Therapeutics and its innovative approaches, visit their website at www.haliatx.com or follow them on LinkedIn and X (Twitter).

In conclusion, the granting of Orphan Drug Designation for ofirnoflast represents a significant step forward in the quest for effective treatments for Myelodysplastic Syndromes. With a focus on addressing the root causes of inflammation, Halia Therapeutics may be on the brink of redefining treatment paradigms in this challenging area of medicine.