AtgamModerate-to-severe aplastic anaemia - first-line in adults without a HLA identical sibling for BMT
New molecular entity
Development and Regulatory status
Feb 21Company previously re-filed using the EU decentralised procedure and a decision is expected in Aug 2021 .
Nov 20EU licensing remains Latvia and Slovenia only .
Jul 19Currently appears to be licensed in Latvia and Slovenia only .
Dec 18Still pre-registration; a decision on whether to recommend approval is expected shortly .
Feb 17Currently pre-registration in the EU. Has been filed via the EU mutual recognition procedure .
Feb 17Atgam is approved in the US for the management of allograft rejection in renal transplant patients and for the treatment of moderate to severe AA in patients who are unsuitable for bone marrow transplantation. For AA the recommended dose regimen is 10-20 mg/kg daily for 8 to 14 days; additional alternate-day therapy up to a total of 21 doses can be administered. Atgam is currently not licensed in the UK but is available on a named patient basis .
Atgam is composed of antibodies that bind a wide variety of proteins on the surface of lymphocytes. Atgam binds to granulocytes, platelets and bone marrow cells. Given as 16 mg/kg/day over 10 days or 20 mg/kg/day over 8 days or 40 mg/kg/day over 4 days.
The incidence of acquired AA in Europe and North America is around 2 per million population per year, and it has a biphasic age distribution, with peaks from 10 to 25 years and >60 years .
Moderate-to-severe aplastic anaemia - first-line in adults without a HLA identical sibling for BMT
new dosing regimen from that approved in the US (16 mg/kg/day over 10 days or 20 mg/kg/day over 8 days or 40 mg/kg/day over 4 days)
Trial or other data
Nov 05PIII (NCT00260689/060034, 06-H-0034) study starts. 120 treatment-naive patients (60 per arm) aged 2-77 years with moderate to severe were randomized to receive either hATG at 40 mg/kg/day for 4 days or anti t-lymphocyte (rabbit rATG) at 3,5mg/Kg/day for 6 months. Each a arm also included ciclosporin at 10mg/Kg/day (15mg/kg/day for children under 12) given in divided doses every 12 hours from day 1 for at least 6 months in both the hATG and rATG groups, with the dose adjusted to maintain through blood levels of 200 to 400 ng/ml. The observed rate of haematologic response at 6 months was in favour of hATG (68%; 95% confidence interval -CI-, 56 to 80%) as compared with rATG (37%; 95% CI, 24 to 49%; p<0,001). The overall survival rate at 3 years differed significantly between the two regimens: 96% (95% CI, 90 to 100%) in the h ATG group as compared with 76% (95% CI, 61 to 95%) in the rATG group (p=0,004) when data were censored at the time of stem cell transplantation, and 94% (95% CI, 88 to 100%) as compared with 70% (95% CI, 56 to 86%; p=0,008) in the respective groups when stem cell transplantation eventes were not censored .