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.

Position paper Unleashing Therapeutic Innovation for Patients By Overcoming Hurdles to New Uses of Authorized drugs

The repurposing of authorized drugs by nontraditional developers that demonstrate new uses for existing medicines through noncommercial research offers a promising complementary path for generating patient benefit and public health impact. It can lead to faster and more cost-effective medical innovation compared to traditional drug development and potentially deliver more affordable treatments. This approach is especially useful for addressing important health needs such as those of patients with rare diseases or paediatric cancers, as these areas often lack commercial interest. It also allows for the exploration of diverse research avenues that were previously overlooked due to a lack of profit incentives. Realizing this potential requires a supportive policy and regulatory framework. The proposed EU Pharmaceutical Regulation, especially Article 48, could be transformative to this effect. It allows non-profit organizations to submit non-clinical and clinical evidence for new uses of authorized drugs to national or European regulators for assessment and provides a pathway to label change. This in turn will facilitate integration of the new use into medical guidelines and reimbursement schemes, enhancing and accelerating the adoption of innovative treatments with clear patient benefit into clinical practice.

Full paper: Anticancer Fund Repurposing Paper 20032025