Research Developments

PROGRESS IN TREATMENT DEVELOPMENT IN BOTH EUROPE AND US ON FOETAL HAEMOGLOBIN BOOSTING THERAPY: IMR-687

The European Commission and US Food and Drug Administration have granted orphan drug designation to a new sickle cell therapy developed by Imara Inc. This designation indicates that the treatment has enough data to back up claims of clinical benefits. It also speeds up the process in which the treatment becomes available in the specified market if approved. Imara is a pharmaceutical company that specialises in producing therapies for sickle cell anaemia. IMR-687 is administered orally and targets a biochemical pathway in the body that suppresses the production of foetal haemoglobin. Usually only present in foetuses and newborns, foetal haemoglobin is a protein in the red blood cells that carries oxygen in the body much like adult haemoglobin. Increase in foetal haemoglobin could reduce the symptoms associated with sickle cell anaemia and beta thalassaemia major, which is what IMR-678 hopes to do. IMR-687 has a long road of clinical studies ahead. It is currently undergoing Phase 2b trials in the US, testing its safety and effectiveness in patients with sickle cell anaemia.

LUSPARTERCEPT APPROVED IN EUROPE

Luspartercept (also branded as Reblozyl), already approved by the Food and Drug Administration in the United States, is a red blood cell boosting drug for the treatment of anaemias including beta thalassaemia. Earlier this year it also had the approval of the European Medicine Agency. Luspartercept interrupts the biochemical pathways that regulate red blood cell maturation and increases red blood cell count. This is expected to reduce the units of blood required by patients over time. A long term surveillance study of 336 patients showed that effects were lower in pts patients???? with the most severe disease forms of beta thalassaemia. However, meaningful reductions in transfusion burden were observed across all patients studied.

Sources:

Carvalho J. European Commission Grants Orphan Drug Status to Imara’s IMR-687... [Internet]. Sickle Cell Disease News. 2020 [cited 28 September 2020]. Available from: https://sicklecellanemianews.com/2020/08/27/ european-commission-grants-orphan-drug-designation-to-imr-687-for-scd/ 2. Reblozyl - European Medicines Agency [Internet]. European Medicines Agency. 2020 [cited 28 September 2020]. Available from: https://www.ema. europa.eu/en/medicines/human/EPAR/reblozyl Cappellini MD, Viprakasit V, Taher AT, Georgiev P, Kuo KH, Coates T, Voskaridou E, Liew HK, Pazgal-Kobrowski I, Forni GL, Perrotta S. A phase 3 trial of luspatercept in patients with transfusion-dependent β-thalassemia. New England Journal of Medicine. 2020 Mar 26;382(13):1219-31.

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