Drug repositioning framework driven by landscape analysis and competitive intelligence: A case report on Prader Willi Syndrome

Drug repositioning is a promising strategy to develop therapeutics for rare diseases ^1,2. In our present work, we explore a drug repositioning framework that can capture the clinical unmet needs of Prader Willi syndrome (PWS) showing ineffectiveness of GLP1 agonists ³, widely accepted as the standard of care for lifestyle obesity. Several therapeutic axes have been explored against PWS such as enzyme replacement therapy ^4,5, Ghrelin analogues ⁶, MC4R agonist ⁷, however none of these approaches have shown preventive action, thus leaving a wide scope for exploring drug repositioning in PWS. Market share analysis of GLP1 agonists used against PWS along with the overall market size and growth of obesity medication shows the room for market penetrance in rare conditions of genetic obesity. Novo Nordisk and Eli Lilly dominate the obesity therapeutics market with their blockbuster drugs like Ozempic, Wegovy ⁸, Trulicity and most recently Zepbound ⁹. In addition, entry of Zealand Pharmaceuticals and Viking Therapeutics are set to provide additional therapeutic options to patients of obesity ^10,11. However, the competitive landscape surrounding PWS therapies points to a significant gap, as genetic obesity has potential multi-systemic involvement and not only the metabolic axis ¹². This framework provides life cycle management opportunities in other genetic obesity conditions such as Bardet-Biedl syndrome (BBS), Kleefstra syndrome- EHMT1 mutation, Alstrom syndrome, Biemond syndrome type 2, Wolfram syndrome, Cohen syndrome, familial isolated DCM and mitochondrial disorders ¹³.

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Publisher: REPO4EU

Publication date (Electronic, pub): 30 April 2024

Affiliations

[1 ] MedInsights SAS, Paris, France;

Author information

Manish Sarkar https://orcid.org/0000-0002-0861-888X

Soham Saha https://orcid.org/0000-0003-1173-1397

Article

DOI: 10.58647/REXPO.24003

SO-VID: bd6bd751-654c-4d3d-a8e4-acaa250b95fa

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Published under Creative Commons Attribution 4.0 International ( CC BY 4.0). Users are allowed to share (copy and redistribute the material in any medium or format) and adapt (remix, transform, and build upon the material for any purpose, even commercially), as long as the authors and the publisher are explicitly identified and properly acknowledged as the original source.

Conference name: RExPO24

Conference number: 3

Conference location: Munich, Germany

Conference date: 3-5 July 2024

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REPO4EU

References

Roessler Helen I., Knoers Nine V.A.M., van Haelst Mieke M., van Haaften Gijs. Drug Repurposing for Rare Diseases. Trends in Pharmacological Sciences. Vol. 42(4):255–267. 2021. Elsevier BV. [Cross Ref]
Jonker Anneliene Hechtelt, O’Connor Daniel, Cavaller-Bellaubi Maria, Fetro Christine, Gogou Maria, ’T Hoen Peter A. C., de Kort Martin, Stone Heather, Valentine Nivedita, Pasmooij Anna Maria Gerdina. Drug repurposing for rare: progress and opportunities for the rare disease community. Frontiers in Medicine. Vol. 11:2024. Frontiers Media SA. [Cross Ref]
Ng Nicholas Beng Hui, Low Yue Wey, Rajgor Dimple Dayaram, Low Jia Ming, Lim Yvonne Yijuan, Loke Kah Yin, Lee Yung Seng. The effects of glucagon‐like peptide (GLP)‐1 receptor agonists on weight and glycaemic control in Prader–Willi syndrome: A systematic review. Clinical Endocrinology. Vol. 96(2):144–154. 2022. Wiley. [Cross Ref]
Cassidy Suzanne B, Driscoll Daniel J. Prader–Willi syndrome. European Journal of Human Genetics. Vol. 17(1):3–13. 2009. Springer Science and Business Media LLC. [Cross Ref]
Allas Soraya, Caixàs Assumpta, Poitou Christine, Coupaye Muriel, Thuilleaux Denise, Lorenzini Françoise, Diene Gwenaëlle, Crinò Antonino, Illouz Frédéric, Grugni Graziano, Potvin Diane, Bocchini Sarah, Delale Thomas, Abribat Thierry, Tauber Maithé. AZP-531, an unacylated ghrelin analog, improves food-related behavior in patients with Prader-Willi syndrome: A randomized placebo-controlled trial. PLOS ONE. Vol. 13(1)2018. Public Library of Science (PLoS). [Cross Ref]
Loos Ruth J. F., Yeo Giles S. H.. The genetics of obesity: from discovery to biology. Nature Reviews Genetics. Vol. 23(2):120–133. 2022. Springer Science and Business Media LLC. [Cross Ref]

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[1] Roessler Helen I., Knoers Nine V.A.M., van Haelst Mieke M., van Haaften Gijs. Drug Repurposing for Rare Diseases. Trends in Pharmacological Sciences. Vol. 42(4):255–267. 2021. Elsevier BV. [Cross Ref]

[2] Jonker Anneliene Hechtelt, O’Connor Daniel, Cavaller-Bellaubi Maria, Fetro Christine, Gogou Maria, ’T Hoen Peter A. C., de Kort Martin, Stone Heather, Valentine Nivedita, Pasmooij Anna Maria Gerdina. Drug repurposing for rare: progress and opportunities for the rare disease community. Frontiers in Medicine. Vol. 11:2024. Frontiers Media SA. [Cross Ref]

[3] Ng Nicholas Beng Hui, Low Yue Wey, Rajgor Dimple Dayaram, Low Jia Ming, Lim Yvonne Yijuan, Loke Kah Yin, Lee Yung Seng. The effects of glucagon‐like peptide (GLP)‐1 receptor agonists on weight and glycaemic control in Prader–Willi syndrome: A systematic review. Clinical Endocrinology. Vol. 96(2):144–154. 2022. Wiley. [Cross Ref]

[4] Cassidy Suzanne B, Driscoll Daniel J. Prader–Willi syndrome. European Journal of Human Genetics. Vol. 17(1):3–13. 2009. Springer Science and Business Media LLC. [Cross Ref]

[5] Allas Soraya, Caixàs Assumpta, Poitou Christine, Coupaye Muriel, Thuilleaux Denise, Lorenzini Françoise, Diene Gwenaëlle, Crinò Antonino, Illouz Frédéric, Grugni Graziano, Potvin Diane, Bocchini Sarah, Delale Thomas, Abribat Thierry, Tauber Maithé. AZP-531, an unacylated ghrelin analog, improves food-related behavior in patients with Prader-Willi syndrome: A randomized placebo-controlled trial. PLOS ONE. Vol. 13(1)2018. Public Library of Science (PLoS). [Cross Ref]

[6] Loos Ruth J. F., Yeo Giles S. H.. The genetics of obesity: from discovery to biology. Nature Reviews Genetics. Vol. 23(2):120–133. 2022. Springer Science and Business Media LLC. [Cross Ref]