US-based clinical-stage biotech Halia Therapeutics, Inc. announced that its investigational treatment ofirnoflast (HT‑6184) has been granted an Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for use in Myelodysplastic Syndromes (MDS), a bone-marrow disorder marked by ineffective blood-cell production and potential progression to acute myeloid leukemia.
The company said the FDA award recognises the unmet need in MDS, a condition affecting fewer than 200,000 people in the US at the time of designation.
Ofirnoflast is described by Halia Therapeutics as a first-in-class NEK7 allosteric modulator aimed at preventing formation and promoting disassembly of the NLRP3 inflammasome, a key driver of chronic inflammation and impaired hematopoiesis in MDS.
The designation provides the company with potential benefits including tax credits for qualified clinical testing, exemption from certain FDA user fees, and the possibility of seven-year U.S. market exclusivity if the drug is approved.
According to the company, exploiting the inflammasome biology in MDS may offer a novel approach compared with treatments that target downstream effects of the disease rather than upstream inflammatory mechanisms.
Halia Therapeutics reported that ofirnoflast is already under evaluation across multiple disease indications including MDS, obesity (in combination with semaglutide) and Alzheimer’s disease. The company claims this designation underscores its strategy to develop therapies addressing root causes of inflammation-driven diseases.
