Repurposing Drugs for Orphan Diseases

Review Article | Volume 3 Issue 1

C Fratazzi* and J Niu
*Corresponding Author: C Fratazzi, BBCR Consulting, One Broadway, Cambridge, MA, USA.

BBCR Consulting, One Broadway, Cambridge, MA, USA

Abstract

Drug repurposing, also known as drug repositioning or reprofiling, develops and commercializes new uses for existing or abandoned drugs. In past decades, ~30% of repurposed drugs were approved by the United States Food and Drug Administration (FDA), while less than 10% of de novo drug applications achieved approval.

Repurposing drugs were developed for various diseases, including cancer, infection, inflammatory disorders, and neurodegenerative diseases. Experimental and computational strategies have been applied in drug repurposing. Professional expert guidance is an essential support for developing a successful regulatory and clinical drug repurposing plan. Our knowledge and experience in regulatory, clinical research, and biomarker consulting provide high-value and support our clients’ operational and functional needs. The opportunity to support clients’ implementing the most appropriate FDA 505(b)(2) and EMA hybrid submissions maximizes the benefit of drug repurposing for orphan indications. It helps patients with rare diseases and the healthcare system.

This review focuses on developing repurposing drugs for orphan indications and rare diseases. Due to the significant complexity of orphan drug development and lack of effective treatment for many of them, we aim to provide options to address the current unmet medical needs for a wide range of patients worldwide.

References

1. Osakwe O and Rizvi S. “Social aspects of drug discovery, development, and commercialization” (2016).
2. Parvathaneni V., et al. “Drug repurposing: a promising tool to accelerate the drug discovery”. Drug Discovery Today10 (2019): 2076-2085.
3. Research and Markets. “Drug repurposing service providers market, 2020-2030” (2020).
4. Murteira S., et al. “Drug reformulations and repositioning in pharmaceutical industry and its impact on market access: reassessment of nomenclature”. Journal of Market Access and Health Policy1 (2013).
5. The US FDA. “Learn about drug and device approvals” (2018).
6. Dhir N., et al. “Drug repurposing and orphan disease therapeutics”. Intech Open Book Series (2020).
7. Li YY and Jones S. “Drug repositioning for personalized medicine”. Genome Medicine 4 (2012): 27.
8. ACCORDING TO A NEW STUDY, Terry M. “The median cost of bringing a drug to market is $985 million”. Biospace (2020).
9. Rudrapal M., et al. “Drug repurposing (DR): An emerging approach in drug discovery”. Intech Open Book Series (2020).
10. Pillaiyar T., et al. “A medicinal chemistry perspective of drug repositioning: Recent advances and challenges in drug discovery”. European Journal of Medicinal Chemistry 195 (2020).
11. Chen H., et al. “Scaffold repurposing old drugs towards new cancer drug discovery”.
12. Current Topics in Medicinal Chemistry19 (2016): 2107-2014.
13. Stewart J. “Actemra FDA approval history” (2021).
14. Ghofrani H., et al. “Sildenafil: from angina to erectile dysfunction to pulmonary hypertension and beyond”. Nature Reviews Drug Discovery 5 (2006): 689-702.
15. Thayer A M. “Drug repurposing: finding new uses for approved drugs and shelved drug candidates is gaining steam as a pharmaceutical development strategy”. 90.40 (2012).
16. Ashburn TT and Thor K B. “Drug repositioning: identifying and developing new uses for existing drugs”. Nature Review Drug Discovery8 (2004): 673-683.
17. Karaman B., et al. “Computational drug repurposing: current Trends”. Current Medicinal Chemistry 28 (2019): 5389-5409.
18. Park K. “A review of computational drug repurposing”. Translational and Clinical Pharmacology 2 (2019): 59-63.
19. Surrogate Endpoints to accelerate orphan drug approval.
20. The US FDA. “Guidance for industry: applications covered by section 505 (b) (2)” (1999).
    “European medicines agency procedural advice for users of the centralized procedure for generic/hybrid applications”. 2021 version.
21. US FDA. “NDA and BLA calendar year approvals”.
22. US FDA, CDER. “Determining whether to submit an ANDA or a 505 (b) (2) application” (2019).
23. Rolland B., et al. “France approves baclofen for alcohol dependence”. Alcohol Alcohol1 (2020): 44-45.
24. Bayoumy A B., et al. “Unrealized potential of drug repositioning in Europe during COVID-19 and beyond: a physician’s perspective”. Expert Opinion on Drug Discovery8 (2021).
25. Gil C and Martinez A. “Is drug repurposing the future of drug discovery, or is innovation indeed the way forward”. Expert Opinion on Drug Discovery8 (2021): 829-831.

Citation

Citation: C Fratazzi and J Niu. “Repurposing Drugs for Orphan Diseases”. Acta Scientific Pharmacology 3.1 (2022): 36-41.
Copyright

Copyright: © 2022 C Fratazzi and J Niu. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

Keywords: Drug repurposing (DR); Food and Drug Administration (FDA); Drugs