New Medicines

Gamifant · Primary haemophagocytic lymphohistiocytosis (HLH)


New molecular entity
Swedish Orphan Biovitrum (Sobi)
Swedish Orphan Biovitrum (Sobi)

Development and Regulatory status

Pre-registration (Filed)
Pre-registration (Filed)
Pre-registration (Filed)

Oct 18: Now reported as not being evaluated in the EU using the accelerated assessment programme [6].

Aug 18: filed in EU with accelerated assessment, for treatment of patients with primary Haemophagocytic Lymphohistiocytosis (HLH). Accelerated assessment cuts 60 days from the standard CHMP timeline, with the potential for a CHMP opinion in less than five months from submission [5].

Mar 18: Filed in US for treatment of patients with primary HLH [3].


Monoclonal antibody, targeting anti-interferon-γ IFNγ
Primary HLH is a clinical syndrome of hyperinflammation, driven by high interferon gamma production and characterised by severe hyperferritinemia, fever, severe cytopenia, coagulation defects and organomegaly. Approximately 1 per 200 000 children become unwell with HLH each year; most (70-80%) become unwell in the first year of life [1].
Primary haemophagocytic lymphohistiocytosis (HLH)

Further information


Trial or other data

Mar 18: PII/III study (NCT01818492) is still recruiting [4].

Jan 13: PII/III study (NCT01818492) to assess the safety, tolerability and efficacy of NI-0501 aimed at controlling disease activity in patients diagnosed with primary haemophagocytic lymphohistiocytosis. The new drug can be administered as the first-line therapy, to patients not previously treated with the current standard of care, or can be given to patients who have either failed or were unable to tolerate the current standard of care. Administration will be on top of a glucocorticosteroid, which is usually part of the current recommended treatment. 32 children aged up to 18 years will be recruited in the US, Germany, Italy, Spain, Sweden and the UK. Collection of primary outcome data (overall response rate) is due to complete Jun 18 [4].

Evidence based evaluations