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Orphan drugs will double their market share by 2030
By 2030, orphan drugs will double their market share to 20% of the $1.6 trillion global drug market, with a CAGR of over 10%, outperforming the 7.5% of non-orphan drugs.
Once specialized treatments for rare conditions, orphan drugs are now challenging the dominance of mainstream medications. Highlighted in two Evaluate reports, "Are Orphans That Different?" and "Top Orphan Drugs: Still a Class Apart?", published on March 14, 2025, these drugs are gradually becoming indistinguishable from conventional pharmaceuticals. As the lines between treatments for rare diseases and traditional therapies blur, a new era in the global drug market seems increasingly likely.
Market growth and trends
Both reports suggest orphan drugs are forecasted to constitute a fifth of the $1.6 trillion in global prescription drug sales by 2030, a share that has impressively doubled over the past decade. The category's compound annual growth rate from 2025 to 2030 is projected at over 10%, outpacing the 7.5% for non-orphan drugs.
Despite this robust growth, the advantage is anticipated to narrow significantly, dropping to just 1% by the decade's end due to increasing competition and market saturation -- challenges that were previously more characteristic of conventional medications.
Top performers
Orphan drugs are increasingly rivaling traditional blockbuster therapies, with several expected to achieve significant market success:
- Darzalex (daratumumab). This monoclonal antibody medication made by Johnson & Johnson (J&J) to treat multiple myeloma is projected to exceed $16 billion in sales by 2030.
- Alyftrek (vanzacaftor/tezacaftor/deutivacaftor). Vertex Pharmaceuticals' cystic fibrosis therapy for patients six or older is predicted to make a major market impact.
- Winrevair (sotatercept). Merck's injectable activin signaling inhibitor treatment for pulmonary arterial hypertension has strong sales projections.
The performance of these drugs suggests orphan drugs are no longer confined to niche markets but are competing directly with mainstream treatments in terms of sales and market penetration.
Pipeline innovation
The orphan drug pipeline remains active, constituting 20% of global pipeline forecast sales. Among the standout developments is anito-cel (anitocabtagene autoleucel), a Phase 3 B-cell maturation antigen-directed CAR-T cell therapy being co-developed by Gilead Sciences and Arcellx. Projected to bring in nearly $1.4 billion by 2030, this developmental treatment modifies patients' immune cells to fight cancer, showing promise for difficult-to-treat forms of relapsed or refractory multiple myeloma.
Corcept Therapeutics is also making waves with its selective cortisol modulator, relacorilant, designed to treat conditions that arise from the body's mismanagement of cortisol, such as Cushing's syndrome.
Regulatory and economic challenges
Meanwhile, the Inflation Reduction Act's impact on high-cost medications like J&J's Darzalex introduces uncertainty in revenue forecasts, with potential price negotiations that could affect profitability. This variability requires companies to be agile in their strategy and pricing models to adapt to new governmental policies that could reshape the market.
The blurring lines between orphan and non-orphan drugs are evident not only in revenue but also in regulatory and market behaviors. As orphan drugs increasingly resemble mainstream pharmaceuticals, whether they should remain a distinct category or become integrated into the broader market must be determined. Ultimately, a convergence could change how these drugs are developed, marketed and regulated.
Industry implications
The integration of orphan drugs into the mainstream market suggests several industry implications. Companies might need to rethink pricing strategies to balance profitability with access, especially under tighter regulatory scrutiny. There could be a shift toward more collaborative research and development efforts, leveraging advances in technology and data sharing to reduce costs and accelerate development timelines.
Additionally, marketing strategies may need adjustment as the target audience for orphan drugs expands beyond niche specialists to general practitioners and broader healthcare systems.
As the distinction between orphan drugs and traditional medications fades, industry stakeholders should reconsider how these drugs fit into the broader healthcare system -- which may require an overhaul in development, marketing and regulation.
Alivia Kaylor is a scientist and the senior site editor of Pharma Life Sciences.
