Gamifant · Primary haemophagocytic lymphohistiocytosis (HLH)
Development and Regulatory status
Dec 18: Launched in the US 
Nov 18: Approved in the US 
Oct 18: Now reported as not being evaluated in the EU using the accelerated assessment programme .
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 .
Mar 18: Filed in US for treatment of patients with primary HLH .·
Trial or other data
Dec 18: Data from the pivotal PII/III study (n=34) presented at the annual meeting of the American Society of Hematology (ASH). The trial achieved its primary endpoint, with 64.7% of all patients treated (22 of 34; p=0.0031) and 63% of the patients who had failed prior conventional HLH therapy (17 of 27; p=0.0134) demonstrating an overall response at the end of treatment, defined as achievement of either a complete or partial response, or HLH improvement. Across both cohorts, the overall response rate (ORR) was significantly higher than the pre-specified null hypothesis of 40%. The median time to response was 8 days, and patients remained in response for a median of 75% of the time.The majority of all patients treated – 64.7% (22 of 34) of the full patient group and 70.4% (19 of 27) of the patients who had failed prior conventional HLH therapy – proceeded to haematopoietic stem cell transplantation (HSCT), with 90.9% (20 of 22) of the full group and 89.5% (17 of 19) of the subgroup surviving post HSCT. The most common adverse reactions reported during the study were infections (56%), hypertension (41%), infusion-related reactions (27%) and fever (24%) .
Mar 18: PII/III study (NCT01818492) is still recruiting .
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 .·