Leading orphan drugs in development based on net present value 2024

Published by
Matej Mikulic,
Dec 10, 2024
This statistic displays a projection of the top five orphan R&D products (phase III/filed) ranked by net present value as of February 2024. Vertex' VX-121 had a net present value of 5.8 billion U.S. dollars. Orphan drugs are used to target rare diseases or disorders, which in the United States is defined as less than 200,000 patients. The development of orphan drugs are currently incentivized in the United States through the Orphan Drug Act of 1983.

Leading orphan drugs in development (phase III/filed) by NPV as of 2024 (in billion U.S. dollars)

CharacteristicNet present value in billion U.S. dollars
VX-121 (Vertex Pharmaceuticals) 5.8
Nipocalimab (Johnson & Johnson) 3.6
NTLA-2001 (Intellia Therapeutics) 3.5
Paltusotine (Crinetics Pharmaceuticals) 3.3
Bemarituzumab (Amgen) 2.7
Showing entries 1 to 5 (5 entries in total)
Created with Highcharts 7.2.2Net present value in billion U.S. dollars5.85.83.63.63.53.53.33.32.72.7VX-121 (Vertex Pharmaceuticals)Nipocalimab (Johnson & Johnson)NTLA-2001 (Intellia Therapeutics)Paltusotine (Crinetics Pharmaceuticals)Bemarituzumab (Amgen)
00.511.522.533.544.555.566.5

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Release date

April 2024

Region

Worldwide

Survey time period

as of February 2024

Supplementary notes

Projected top orphan drugs in 2028, ranked by NPV (net present value) as of February 2024.

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