Anti Cancer Fund

Sorafenib, an off-patent drug, indicated for the treatment of unresectable hepatocellular carcinoma, advanced renal cell carcinoma & differentiated thyroid cancer, has been widely investigated and proven effective as post-transplant treatment in FLT3-ITD-positive AML.

Despite its solid pre-clinical and clinical evidence, broadly published in literature and uptake in several guidelines (amongst others NCCN and EU Society for Blood & Marrow Transplantation), the additional indication hasn’t been requested by the Market Authorization Holder, hence not approved by regulators and is therefore not on-label.

We strive to explore solutions to extend the label, be it through scientific advice, attempting a type II variation with one of the Marketing Authorization Holders, and/or other pathways (EU HTA, upcoming revised Pharma Legislation, …) to ensure all patients across Europe can get access to this effective treatment.