Drug Repurposing for Orphan Disease

Authors

  • Hemant Kumar Author
  • Ruhit Ashraf Desh Bhagat University image/svg+xml Author
  • Hema Rani Author

DOI:

https://doi.org/10.64261/s49q9c13

Keywords:

Repurposing Orphan drugs, reprofiling, neurodegenerative

Abstract

There is a great need and opportunity to find treatments for orphan or rare diseases. Of the roughly 6000 orphan diseases, some have little commercial prospects and/or low prevalence. Many chemicals have the potential to be reprofiled in a new indication, even if only a small number of compounds have been licensed for new indications in the field of metabolic diseases. In general, there are three categories into which reprofiled medications for metabolic diseases can be divided. Drug repurposing is the term used to describe the current change in focus to alternative uses for costly new medications. The discovery of novel therapeutic uses for already available or experimental medications is known as drug repurposing or reprofiling. Numerous potentially fatal illnesses, including some types of cancer, neurological problems, immunological disorders, and rare infectious diseases, may benefit from this idea and innovative treatment methods. Numerous potentially fatal illnesses, including some types of cancer, neurological problems, immunological disorders, and rare infectious diseases, may benefit from this idea and innovative treatment methods.

Author Biographies

  • Hemant Kumar

    B Pharm (Student), S. Lal Singh Memorial College of Pharmacy

  • Hema Rani

    Associate Professor, S. Lal Singh Memorial College of Pharmacy

References

[1] S. Pushpakom, F. Iorio, P.A. Eyers, K.J. Escott, S. Hopper, A. Wells, A. Doig, T. Guilliams, J. Latimer, C. McNamee, A. Norris, P. Sanseau D. Cavalla, M. Pirmohamed, Drug repurposing: progress, challenges and recommendations, Nat. Rev. Drug Discover,18 (2018) 41-58, https://doi.org/10.1038/nrd.2018.16.

[2] Ferreira CR. The burden of rare diseases. Am J Med Genet A. 2019; 179:885-92. doi: 10.1002/ajmg.a.61124.

[3] Cremers S, Aronson JK. Drugs for rare disorders. Br J Clin Pharmacol. 2017; 83(8): 1607-1613. 6 Ministry of Health, Labour and Welfare of Japan. Designated Intractable Diseases (in Japanese). Ministry of Health, Labour and Welfare of Japan.

[4] J.L. Dahlin, D.S. Auld, I. Rothenaigner, S. Haney, J.Z. Sexton, J.W.M. Nissinket al., Nuisance compounds in cellular assays, Cell Chem Biol 28 (2021) 356–370. 7 J.C. Saiz, M.A. Martín-Acebes, The race to find antivirals for zika virus, Antimicrob Agents Chemother 61 (2017) e00411–e417.

[5] M.S. Kinch et al.Lost medicines: a longer view of the pharmaceutical industry with the potential to reinvigorate discovery Drug Discover Today (2019)

[6] Kulkarni NS et al. (2019) Tyrosine kinase inhibitor conjugated quantum dots for non-small cell lung cancer (NSCLC) treatment. Eur. J. Pharm. Sci. 133, 145–159 [PubMed: 30946965]

[7] Pahud D et al. (2014) A new market access path for repurposed drugs. Available at:2014 In: https://www.kauffman.org/what-we-do/research/2014/05/a-new-market-access-path-for-repurposed-drugs

[8] Kulkarni, V. S., Alagarsamy, V., Solomon, V. R., Jose, P. A., & Murugesan, S. (2023). Drug repurposing: an effective tool in modern drug discovery. Russian journal of bioorganic chemistry, 49(2), 157-166.

[9] Musyuni, P., Sharma, R., & Aggarwal, G. (2023). Optimizing drug discovery: An opportunity and application with reverse translational research. Health Sciences Review, 6, 100074.

[10] Chen, K., Lim, T. P., Yuen, J. S., Ho, H., & Murphy, D. G. (2025). Regulatory considerations for new drugs and devices. In Translational Urology (pp. 293-299). Academic Press.

[11] Zajki-Zechmeister, T. (2022). A regulatory guide for medical device start-ups in Europe: challenges and pitfalls. In Medical Devices and In Vitro Diagnostics: Requirements in Europe (pp. 1-25). Cham: Springer International Publishing.

[12] Aartsma-Rus, A., Dooms, M., & Le Cam, Y. (2021). 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, 12, 744532.

[13] Dang, K. X. (2026). Death By A Thousand Cuts: Patent Litigation Risks and their Chilling Effects on Pharmaceutical Investments and Innovation in the United States. Houston Journal of Health Law & Policy, 25(2).

[14] Harper, P. (2019). The evolution of medical genetics: a British perspective. CRC Press.

[15] Ranganath, L. R., Psarelli, E. E., Arnoux, J. B., Braconi, D., Briggs, M., Bröijersén, A., ... & Gallagher, J. A. (2020). Efficacy and safety of once-daily nitisinone for patients with alkaptonuria (SONIA 2): an international, multicentre, open-label, randomised controlled trial. The Lancet Diabetes & Endocrinology, 8(9), 762-772.

[16] Genovese F, Frederiksen P, Bay-Jensen A-C, et al. Nitisinone treatment affects biomarkers of bone and cartilage remodelling in alkaptonuria patients. Int J Mol Sci. 2023;24(13):10996

[17] Previtali R, Prontera G, Alfei E, et al. Paradigm shift in the treatment of tuberous sclerosis: effectiveness of everolimus. Pharmacol Res. 2023,195:106884.

[18] Britti, E., Delaspre, F., Feldman, A., Osborne, M., Greif, H., Tamarit, J., et al. (2018). Frataxin-deficient Neurons and Mice Models of Friedreich Ataxia Are Improved by TAT-MTScs-FXN Treatment. J. Cel Mol Med 22, 834–848. doi:10.1111/jcmm.13365 Buyse, G., Mertens, L., Di Salvo, G., Matthijs, I., Weidemann, F., Eyskens, B., et al. (2003). Idebenone Treatment in Friedreich’s Ataxia: Neurological, Cardiac, and Biochemical Monitoring. Neurology 60, 1679–1681. doi:10.1212/ 01.wnl.0000068549.52812.0f

[19] Krishnamurthy N, Grimshaw AA, Axson SA, et al. Drug repurposing: a systematic review on root causes, barriers and facilitators. BMC Health Serv Res. 2022;22(1):970.

[20] De Rosa MC, Purohit R, García-Sosa AT. Drug repurposing: a nexus of innovation, science, and potential. Sci Rep. 2023;13(1):17887

[21] Mayack BK, Sippl W. Current In silico drug repurposing strategies. Syst Med. 2021;257–68

[22] De Simone G. Sardina DS, Gulotta MR, Perricone U. KUALA: a machine learning-driven framework for kinase inhibitors repositioning. Sci Rep. 2022;12(1):17877.

Downloads

Published

04.04.2026

How to Cite

Drug Repurposing for Orphan Disease. (2026). Pan-African Journal of Health and Psychological Sciences, 2(2). https://doi.org/10.64261/s49q9c13

Most read articles by the same author(s)

<< < 1 2 

Similar Articles

You may also start an advanced similarity search for this article.