The legislative reform being prepared by the EU aims to promote the use of already approved drugs for new conditions. This approach allows for speeding up the approval of treatments and saving costs on research. 

Nick Sireau, a 27-year-old aid worker, discovered a dark reddish stain in his newborn son’s diaper back in October 2000. It turned out to be a symptom of a rare genetic disorder, alkaptonuria, which also affected Sireau’s second son, manifesting in darkened tissues and spinal arthritis. Nearly two decades later, on September 17, 2020, the European Medicines Agency (EMA) approved the first treatment for alkaptonuria, a drug called nitisinone, which had long been used for another condition. The approval was the result of Sireau’s tireless research efforts to find a cure for his children. The repurposing of existing drugs for new conditions, a practice the EU plans to promote, holds enormous potential, especially for treating the 7,000 described rare diseases, less than a thousand of which have a pharmacological treatment. The process of EMA approval, however, is not easy due to stringent technical requirements and a lack of incentives. Experts suggest that creating a third category of medicines, between innovative new therapies and cheap generics, could encourage investment in repurposed drugs. Additionally, facilitating academic research access to EMA approvals could also help in this regard. The EU’s ongoing legislative reform intends to establish a framework for repurposing drugs, allowing for the convergence of industry and academia. During a recent conference in Barcelona, successful drug repurposing stories were presented, including the use of anastrozole as a preventive measure against breast cancer in high-risk postmenopausal women, and the potential of nitroxoline, an antimicrobial, in treating a rare type of encephalitis.