Health Care & Life Sciences » Pharmaceuticals » Orphan drugs | Top 10 orphan drugs in development by NPV 2018

Leading orphan drugs in development based on net present value 2018

Projection of top 10 orphan drugs in development (phase III/filed) by NPV as of 2018 (Figures expressed in millions of U.S. dollars, unless otherwise specified)
Created with Highcharts 5.0.147 4807 4805 4585 4585 0535 0534 7494 7494 5764 5764 4774 4773 4503 4503 4003 4002 8812 8812 7882 788Lanadelumab (Shire)AVXS-101 (AveXis)Luspatercept (Celgene)LentiGlobin (Bluebird Bio)Valoctocogene Roxaparvovec (BioMarin)Patisiran (AInylam)Encorafenib (Array BioPharma)MultiStem (Athersys)NuThrax (Emergent BioSolutions)Gilteritinib (Astellas)0500100015002000250030003500400045005000550060006500700075008000

The data shows a projection of the top 10 orphan R&D products (phase III/filed) by net present value as of 2018. Celgene's Luspatercept had a net present value of 5.05 billion USD. Orphan drugs are used to target rare diseases or disorders, which in the U.S. is defined as less than 200,000 patients. The development of orphan drugs are currently incentivized in the U.S. through the Orphan Drug Act of 1983.

Lanadelumab (Shire) 7480
AVXS-101 (AveXis) 5458
Luspatercept (Celgene) 5053
LentiGlobin (Bluebird Bio) 4749
Valoctocogene Roxaparvovec (BioMarin) 4576
Patisiran (AInylam) 4477
Encorafenib (Array BioPharma) 3450
MultiStem (Athersys) 3400
NuThrax (Emergent BioSolutions) 2881
Gilteritinib (Astellas) 2788

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