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# FDA Grants Novimmune Breakthrough Status for NI-0501
- URL: https://www.fdaweb.com/fda-grants-novimmune-breakthrough-status-for-ni-0501/
- Published: 2016-03-16T12:00:00.000Z
- Updated: 2026-09-15T02:44:20.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5135106

> FDA has granted Novimmune a breakthrough therapy designation for NI-0501 and its use in treating patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory disease, or with recurrent or progressive disease during conventional therapy. The therapy is a fully human, anti-interferon-gamma monoclonal antibody that received an orphan drug designation from the agency in 2010\. The breakthrough designation was granted based on clinical data from a Phase 2 study in children with primary HLH, which is a “hyperinflammatory syndrome characterized by uncontrolled and aberrant activation of the immune system and a life-threatening cytokine storm presenting with non-remitting fever, pancytopenia, coagulopathy, and hemophagocytosis potentially leading to death,” according to the company.