Drug Repurposing & repositioning  Stories

Fresh perspectives on how medicines are reimagined
Drug repurposing provides a powerful approach to discovering new therapeutic applications for existing medications, thereby accelerating innovation while reducing development time and costs. This collection of Drug Repurposing Stories showcases successful and ongoing cases that illustrate the many paths to repurposing — from serendipitous discoveries to data-driven approaches.
Each story offers a deeper look into the scientific, regulatory and collaborative processes that turn known molecules into new treatments. By revisiting what we already know, researchers can unlock fresh possibilities for patients and make biomedical research more efficient, sustainable and impactful.
The Drug Repurposing Stories shares a recent repurposing story every month on social media. These approved or prospective repurposing cases are then combined into a quarterly newsletter with a few more interesting cases and extra information. The first edition of the Medicines Reimagined Newsletter is coming soon!
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SOTATERCEPT (WINREVAIR)

From blood disorders to pulmonary hypertension

Sotatercept is a recombinant fusion protein that traps activin ligands to rebalance signalling pathways linked to cell growth. Initially developed for bone loss and later for anaemia in β-thalassemia, its early programs were discontinued.

Years later, promising preclinical results were followed by Phase II and Phase III trials, leading to its repositioning for pulmonary arterial hypertension (PAH).

View the summary here.

References

  • Sotatercept for the treatment of pulmonary arterial hypertension. New England Journal of Medicine. 2021; 384(13):1204–1215. doi: 10.1056/NEJMoa2024277.

  • Sotatercept increases bone formation and bone mass in postmenopausal women with low bone mass. Journal of Bone and Mineral Research. 2009; 24(4): 744–752. doi.org/10.1359/jbmr.081208

  • Acceleron announces preclinical results in pulmonary arterial hypertension at the American Heart Association 2017 Scientific Sessions. BioSpace. Published November 14, 2017. Available at: https://www.biospace.com/acceleron-announces-preclinical-results-in-pulmonary-arterial-hypertension-at-the-american-heart-association-2017-scientific-sessions

  • Pulmonary arterial hypertension. Genetic and Rare Diseases Information Center (GARD), National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH). Available at: https://rarediseases.info.nih.gov/diseases/7501/pulmonary-arterial-hypertension

  • Merck completes acquisition of Acceleron Pharma Inc. Merck & Co., Inc. Published November 22, 2021. Available at: https://www.merck.com/news/merck-completes-acquisition-of-acceleron-pharma-inc/

  • Pulmonary arterial hypertension: Updates in epidemiology and evaluation of patients. The American Journal of Managed Care (AJMC). Published March 12, 2021. Available at: https://www.ajmc.com/view/pulmonary-arterial-hypertension-updates-in-epidemiology-and-evaluation-of-patients

  • Pulmonary arterial hypertension. Orphanet. Orpha code: 422. Available at: https://www.orpha.net/en/disease/detail/422

  • Pulmonary arterial hypertension. Genetic and Rare Diseases Information Center (GARD), National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH). Available at: https://rarediseases.info.nih.gov/diseases/7501/pulmonary-arterial-hypertension