Novimmune, announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to NI-0501 for the treatment of patients with primary HLH with refractory disease, or with recurrent or progressive disease during conventional therapy. NI-0501 is a fully human, anti-interferon-gamma (IFNγ) monoclonal antibody discovered and developed by Novimmune.
The biological activity of IFNγ, which is considered to have a pivotal pathogenic role in HLH, is neutralized by NI-0501, the first targeted therapy for this disease.
This Breakthrough Therapy Designation for NI-0501 was granted by the FDA on the basis of clinical data from a Phase 2 study in children with primary HLH.
Preliminary results from the Phase 2 study have been presented as a late breaking abstract at the American Society of Hematology meeting in Orlando, 8th December 2015. NI-0501 received orphan drug designation from both the FDA and the EMA in 2010.
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