International Drug Repurposing Patent Landscaping, Part 1: Rare Diseases

In this first installment of a series of papers on landscaping of drug repurposing patents we present an analysis in the context of rare diseases. Using the RepoScope drug repurposing database, developed as part of the REPO4EU Horizon Europe project, we identified 190 documents that were published through the international PCT patent system from 2010 to 2023. These documents claimed utility in a total of 98 rare diseases. We provide detailed tabulated summaries for two representative disease classes: lysosomal storage diseases and muscular dystrophies. High variability in active pharmaceutical ingredients indicated high levels of innovation. Seventy-five percent of rare disease drug repurposing patents were filed by small/medium pharmaceutical companies or universities, often university spin-offs. Big pharmaceutical companies show limited interest in this area, as reflected in their low representation. Approximately one-third of all patent disclosures came from the United States and the European Union. The EU demonstrated a stronger position in patenting for rare diseases than in general pharmaceutical patenting. Patentees from China, Korea, and Japan, who are notable in the general pharmaceutical field, showed limited interest in rare disease drug repurposing patents, in contrast to their publication record in this area.