Rami Levin, President of North America for Swedish Orphan Biovitrum (SOBI) discusses his company’s recent FDA approval, Sobi’s commitment to the rare disease community, and how Sobi is growing its presence in North America.
We sat down with Rami Levin at this year’s American Society of Hematology (ASH) conference where SOBI and Novimmune SA presented data from the pivotal phase 2/3 clinical study of emapalumab-lzsg in primary haemophagocytic lymphohistiocytosis (HLH).
Primary HLH is an aggressive hyper-inflammatory syndrome that can quickly become fatal if not treated. This study demonstrated that emapalumab treatment induced rapid and sustained responses, helping these fragile and often very young patients control HLH activity and reach stem cell transplant, which is the only cure for this devastating disease, potentially without the side effects of prolonged chemotherapy. Further, the 90 per cent survival post-transplant in this study is very encouraging, particularly given the high morbidity and mortality associated with primary HLH.
The trial achieved its primary endpoint, with 64.7 per cent 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 eight 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%).