Pharmaceutical market insight specialist Evaluate has debuted its Orphan Drug Report for 2025, which highlights the recent growth of the sector.
The report, which forecasts the sector's trajectory during the next five years, projects that orphan drugs will make up at least 20% of the $1.6tr that prescription drug sales will likely rake in by 2030.
Evaluate also ntoes that orphan drugs will continue to outpace other niches in the pharmaceutical market for the next few years — though its current growth advantage is narrowing; with the sector forecast to be ahead by just 1% at the end of the decade.
Currently, the regulatory and exclusivity advantages that come with rare disease therapeutic dvelopment are driving growth, and many orphan drugs have become "pharmaceutical blockbusters".
The increase in interest, according to Evaluate, is also linked to the growing awareness of uncommon diseases.
The forecaster also predicts that orphan drug candidates will make up one fifth of the global pipeline forecast sales during the rest of the 2020s.
“Drugs for rare diseases now occupy a significant share of the overall drugs market” said Andreas Hadjivasiliou, Manager, Content Strategy at Evaluate.
“Their increasing similarities to mainstream drugs may mean that they are just as heavily impacted by shifting policy and regulatory changes as the rest of the industry”.
Top sellers
Orphan drugs offer a significant opportunity for developers, as the orphans' growth continues to outpace mainstream drugs.
Notably, the category's forecasted CAGR tops 10%, while non-orphan prescription drugs stop at 7.5%.
Evaluate has forecasting that the top selling orphan drugs in 2030 will be Johnson & Johnson's Darzalex, a monoclonal antibody treatment for multiple myeloma — which is likely to exceed $16bn in annual sales. This makes J&J top seller of orphan drugs globally, with a predicted $31.7bn in sales overall.
Second on the list is Alyftrek, a cystic fibrosis medication produced by Vertex Pharmaceuticals that may reach sales of $8.3bn.
This is closely followed by Merck's hypertension therapeutic Winrevair, which is estimated to rack in $6.6bn by 2030.