Novartis’ Sabatolimab Treatment is Granted Orphan Drug Status by the European Commission

The new drug, sabatolimab (MBG453), manufactured by Novartis to treat myelodysplastic syndromes (MDS) has just been granted orphan drug status by the European Commission.

An experimental immuno-myeloid treatment, sabatolimab attaches to a new target called TIM-3, which is expressed on immune and leukemic cells and not on regular hematopoietic stem cells. Acting on TIM-3 revives the immune system to specifically target leukemic stem cells, the source of MDS and acute myeloid leukaemia (AML), with the potential to offer a long-lasting response.

Clinical data demonstrating an increased response rate in high-risk MDS patients who received sabatolimab plus hypomethylating agents (HMAs) has given the EC the confidence it needed to grant the drug the orphan status and give it the go ahead to be prescribed for treatments.

The designation follows hot on the heals of the US Food and Drug Administration which in May granted sabatolimab fast track status (which aids in the development and accelerates review of treatment of serious conditions and address unmet medical need) to treat adult MDS patients at high- or very high-risk, in combination with HMAs.

In June, the FDA also granted breakthrough therapy designation to Novartis’ 177Lu-PSMA-617, which is designed to treat metastatic castration-resistant prostate cancer.