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.

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Author and article information

Journal

Title: DrugRxiv

Publisher: REPO4EU

Publication date (Electronic preprint): 2 April 2024

Affiliations

[1 ] H.M. Pharma Consultancy;

Author notes

[* ]Email: h.mucke@ 123456hmpharmacon.com .

Author information

Hermann Mucke https://orcid.org/0000-0002-1491-6250

Article

DOI: 10.58647/DRUGARXIV.PR000009.v1

SO-VID: 78ea5fc0-1d4e-4765-bde1-830c2aee768c

License:

This work has been published open access under Creative Commons Attribution License CC BY 4.0 , which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. Conditions, terms of use and publishing policy can be found at www.scienceopen.com .

History

Date received : 2 April 2024

Funding

Funded by: funder-id http://dx.doi.org/10.13039/100018696, HORIZON EUROPE Health;

Award ID: 101057619

References

Aartsma-Rus Annemieke, Dooms Marc, Le Cam Yann. Orphan Medicine Incentives: How to Address the Unmet Needs of Rare Disease Patients by Optimizing the European Orphan Medicinal Product Landscape Guiding Principles and Policy Proposals by the European Expert Group for Orphan Drug Incentives (OD Expert Group). Frontiers in Pharmacology. Vol. 12:2021. Frontiers Media SA. [Cross Ref]
Mucke Hermann A.M., Mucke Eva. Sources and Targets for Drug Repurposing: Landscaping Transitions in Therapeutic Space. ASSAY and Drug Development Technologies. Vol. 13(6):319–324. 2015. Mary Ann Liebert Inc. [Cross Ref]

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[1] Aartsma-Rus Annemieke, Dooms Marc, Le Cam Yann. Orphan Medicine Incentives: How to Address the Unmet Needs of Rare Disease Patients by Optimizing the European Orphan Medicinal Product Landscape Guiding Principles and Policy Proposals by the European Expert Group for Orphan Drug Incentives (OD Expert Group). Frontiers in Pharmacology. Vol. 12:2021. Frontiers Media SA. [Cross Ref]

[2] Mucke Hermann A.M., Mucke Eva. Sources and Targets for Drug Repurposing: Landscaping Transitions in Therapeutic Space. ASSAY and Drug Development Technologies. Vol. 13(6):319–324. 2015. Mary Ann Liebert Inc. [Cross Ref]