Orphan drugs sales share among top pharma companies 2030 forecast

It is projected that in 2030 Vertex will generate more than ** percent of its total sales from orphan drugs. The development of orphan drugs is incentivized in the United States through the Orphan Drug Act of 1983.

Orphan drugs overview

Orphan drugs are pharmaceutical products designed to help treat, prevent and even diagnose extremely rare disorders and disease. A rare disease and disorder is defined as an illness that affects one in ***** people (in Europe) or one that affects less than ******* individuals (in the U.S.). It is projected that by 2030 the top orphan drug, by revenue, will be Darzalex.

The orphan drug market

The number of orphan drugs on the market in the U.S. had increased dramatically over the last two decades. In the U.S., there were over *** drugs accepted with an orphan designation in 2024. Orphan drugs are typically significantly more expensive than non-orphan drugs. The average per patient cost of an orphan drug in the U.S. is about *** times as expensive as a non-orphan drug.