REFERENCES

1. Murteira S, Ghezaiel Z, Karray S, Lamure M. Drug reformulations and repositioning in pharmaceutical industry and its impact on market access: reassessment of nomenclature. J Mark Access Health Policy 2013;1:21131.

2. Mucke HAM. COVID-19 and the drug repurposing tsunami. Assay Drug Dev Technol 2020;18:211-4.

3. Galindez G, Matschinske J, Rose TD, et al. Lessons from the COVID-19 pandemic for advancing computational drug repurposing strategies. Nat Comput Sci 2021;1:33-41.

4. European Medicines Agency. Orphan medicinal product designation 2000-2020. Available from: https://www.ema.europa.eu/en/documents/other/orphan-medicines-figures-2000-2020_en.pdf [Last accessed on 7 Apr 2023].

5. European Medicines Agency. Support for development of orphan medicines. Available from: https://www.ema.europa.eu/en/documents/report/report-workshop-support-orphan-medicines-development_en.pdf [Last accessed on 7 Apr 2023].

6. Southall NT, Natarajan M, Lau LPL, et al. IRDiRC Data Mining and Repurposing Task Force. The use or generation of biomedical data and existing medicines to discover and establish new treatments for patients with rare diseases - recommendations of the IRDiRC Data Mining and Repurposing Task Force. Orphanet J Rare Dis 2019;14:225.

7. Begley CG, Ashton M, Baell J, et al. Drug repurposing: misconceptions, challenges, and opportunities for academic researchers. Sci Transl Med 2021;13:eabd5524.

8. Shim JS, Liu JO. Recent advances in drug repositioning for the discovery of new anticancer drugs. Int J Biol Sci 2014;10:654-63.

9. Ghofrani HA, Osterloh IH, Grimminger F. Sildenafil: from angina to erectile dysfunction to pulmonary hypertension and beyond. Nat Rev Drug Discov 2006;5:689-702.

10. Jourdan JP, Bureau R, Rochais C, Dallemagne P. Drug repositioning: a brief overview. J Pharm Pharmacol 2020;72:1145-51.

11. Barnett CF, Machado RF. Sildenafil in the treatment of pulmonary hypertension. Vasc Health Risk Manag 2006;2:411-22.

12. Roy S, Kloner RA, Salloum FN, Jovin IS. Cardiac effects of phosphodiesterase-5 inhibitors: efficacy and safety. Cardiovasc Drugs Ther 2021;Online ahead of print.

13. Pan Y, Cheng T, Wang Y, Bryant SH. Pathway analysis for drug repositioning based on public database mining. J Chem Inf Model 2014;54:407-18.

14. Pagliazzi A, Oranges T, Traficante G, et al. PIK3CA-related overgrowth spectrum from diagnosis to targeted therapy: a case of CLOVES syndrome treated with Alpelisib. Front Pediatr 2021;9:732836.

15. Scherman D, Fetro C. Drug repositioning for rare diseases: knowledge-based success stories. Therapie 2020;75:161-7.

16. Chang DY, Ma WL, Lu YS. Role of Alpelisib in the treatment of PIK3CA-mutated breast cancer: patient selection and clinical perspectives. Ther Clin Risk Manag 2021;17:193-207.

17. Madsen RR, Vanhaesebroeck B, Semple RK. Cancer-associated PIK3CA mutations in overgrowth disorders. Trends Mol Med 2018;24:856-70.

18. Venot Q, Blanc T, Rabia SH, et al. Targeted therapy in patients with PIK3CA-related overgrowth syndrome. Nature 2018;558:540-6.

19. Canaud G, López Gutiérrez J, Irvine A, et al. LBA23 EPIK-P1: retrospective chart review study of patients (pts) with PIK3CA-related Overgrowth Spectrum (PROS) who have received alpelisib (ALP) as part of a compassionate use programme. Ann Oncol 2021;32:S1297.

20. Rosenblatt M. How academia and the pharmaceutical industry can work together: the president’s lecture, annual meeting of the American Thoracic Society, San Francisco, California. Ann Am Thorac Soc 2013;10:31-8.

21. den Berg S, de Visser S, Leufkens HGM, Hollak CEM. Drug repurposing for rare diseases: a role for academia. Front Pharmacol 2021;12:746987.

22. Hernandez JJ, Pryszlak M, Smith L, et al. Giving drugs a second chance: overcoming regulatory and financial hurdles in repurposing approved drugs as cancer therapeutics. Front Oncol 2017;7:273.

23. Fetro C, Scherman D. Drug repurposing in rare diseases: myths and reality. Therapie 2020;75:157-60.

24. Bharadwaj PR, Bates KA, Porter T, et al. Latrepirdine: molecular mechanisms underlying potential therapeutic roles in Alzheimer’s and other neurodegenerative diseases. Transl Psychiatry 2013;3:e332.

25. Prinz F, Schlange T, Asadullah K. Believe it or not: how much can we rely on published data on potential drug targets? Nat Rev Drug Discov 2011;10:712.

26. WHO-EURO. Repurposing of medicines - the underrated champion of sustainable innovation: policy brief. Available from: https://apps.who.int/iris/handle/10665/342567 [Last accessed on 7 Apr 2023].

27. Cha Y, Erez T, Reynolds IJ, et al. Drug repurposing from the perspective of pharmaceutical companies. Br J Pharmacol 2018;175:168-80.

28. Conour J. ; 0. Why method of treatment patents for repurposed drugs are worth the investment. Available from: https://www.jdsupra.com/legalnews/why-method-of-treatment-patents-for-92813/ [Last accessed on 7 Apr 2023].

29. Verbaanderd C, Rooman I, Huys I. Exploring new uses for existing drugs: innovative mechanisms to fund independent clinical research. Trials 2021;22:322.

30. Léauté-Labrèze C, Dumas de la Roque E, Hubiche T, Boralevi F, Thambo JB, Taïeb A. Propranolol for severe hemangiomas of infancy. N Engl J Med 2008;358:2649-51.

31. Drolet BA, Frommelt PC, Chamlin SL, et al. Initiation and use of propranolol for infantile hemangioma: report of a consensus conference. Pediatrics 2013;131:128-40.

32. U.S. Department of Health and Human Services; Food and Drug Administration. Best practices in developing proprietary names for human prescription drug products guidance for industry. Available from: https://www.fda.gov/media/88496/download [Last accessed on 7 Apr 2023].

33. Léauté-Labrèze C, Hoeger P, Mazereeuw-Hautier J, et al. A randomized, controlled trial of oral propranolol in infantile hemangioma. N Engl J Med 2015;372:735-46.

34. Phillips RJ, Penington AJ, Bekhor PS, Crock CM. Use of propranolol for treatment of infantile haemangiomas in an outpatient setting. J Paediatr Child Health 2012;48:902-6.

35. European Medicines Agency. Paediatric-use marketing authorisations. Available from: https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/paediatric-medicines/paediatric-use-marketing-authorisations [Last accessed on 7 Apr 2023].

36. Hachem M, Bonamonte D, Diociaiuti A, Mantuano M, Teruzzi C. Cost-utility analysis of propranolol versus corticosteroids in the treatment of proliferating infantile hemangioma in Italy. PharmacoEcon Ital Res Artic 2015:17.

37. Jadeja N. The role of IP in strategies for repurposing medicines. Available from: http://www.pinsentmasons.com/out-law/analysis/the-role-of-ip-strategies-repurposing-medicines [Last accessed on 7 Apr 2023].

38. Veldman A, Richter E, Hacker C, Fischer D. The use of off-label medications in newborn infants despite an approved alternative being available-results of a national survey. Pharmacy (Basel) 2022;10:19.

39. Ahmed F, Fattani MT, Ali SR, Enam RN. Strengthening the bridge between academic and the industry through the academia-industry collaboration plan design model. Front Psychol 2022;13:875940.

40. European Commission. Commission Expert Group on safe and timely access to medicines for patients (“STAMP”). Available from: https://health.ec.europa.eu/medicinal-products/pharmaceutical-committee-veterinary-pharmaceutical-committee-and-expert-groups/commission-expert-group-safe-and-timely-access-medicines-patients-stamp_en [Last accessed on 7 Apr 2023].

41. European Medicines Agency. Proposal for a framework to support not-for-profit organisations and academia (institutions and individuals) in drug repurposing. Available from: https://www.ema.europa.eu/en/documents/other/question-answers-repurposing-pilot-project-proposal-framework-support-not-profit-organisations_en.pdf [Last accessed on 7 Apr 2023].

42. European Medicines Agency. Building a European innovation platform for the repurposing of medicinal products. Available from: https://ec.europa.eu/info/funding-tenders/opportunities/portal/screen/opportunities/topic-details/horizon-hlth-2021-disease-04-02 [Last accessed on 7 Apr 2023].

43. Hechtelt Jonker A, Hivert V, Gabaldo M, et al. Boosting delivery of rare disease therapies: the IRDiRC Orphan Drug Development Guidebook. Nat Rev Drug Discov 2020;19:495-6.

44. Deplanque D, Fetro C, Ferry A, et al. Drug repurposing: from the discovery of a useful pharmacological effect to making the treatment available to the patient. Therapie 2023;78:10-8.

45. Innovative health initiative. Available from: https://www.ihi.europa.eu/about-ihi [Last accessed on 7 Apr 2023].

Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
Follow Us

Portico

All published articles are preserved here permanently:

https://www.portico.org/publishers/oae/

Portico

All published articles are preserved here permanently:

https://www.portico.org/publishers/oae/