A Safe(r) Passage Amid Stormy Waters?
Orphan drug developers face a temperamental FDA and must battle for attention with blockbusters in obesity and other common diseases. But orphan drug sales trajectories remain strong, buoyed by important policy wins.
By Melanie Senior, with data analysis by Andreas Hadjivasiliou
Despite policy and pricing turmoil across the pharma industry, sales of orphan drugs continue to rise steadily. They will account for over 21% of all prescription pharmaceutical sales by 2032, according to Evaluate forecasts, up from 15% in 2022. Drugs for rare diseases will generate over $400 billion of the almost $1.9 trillion in projected global prescription drug sales in 2032 – about the same as the entire prescription medicines market twenty years ago.
Orphan drugs will account for over 21% of all prescription pharmaceutical sales by 2032.
Figure 1: Worldwide Orphan Drug Sales & Share of Prescription Drug Market (2022-32)
The top eight orphans will each sell over $6 billion worldwide in 2032. Category leader Darzalex (daratumumab), for multiple myeloma, will reach almost twice that thanks in part to a sub-cutaneous formulation, which doubles the franchise’s effective lifespan. An orphan-friendly tweak to the Inflation Reduction Act (IRA) legislation in 2025 also helps.
Figure 2: Top 10 Selling Orphan Drugs in 2032 by Worldwide Sales
Darzalex secures sponsor Johnson & Johnson’s position a-top the biggest companies by orphan drug sales, with almost $31 billion in projected orphan drug sales in 2032 (40% of total sales). Gate-crashing the top ten companies that year: Dutch-incorporated Argenx, forecast to displace Pfizer in the orphan drug sales rankings. Argenx is seeking to emulate Darzalex’s trajectory with Vyvgart (efgartigimod alfa) and subcutaneous Vyvgart Hytrulo in myasthenia gravis and other rare auto-immune disorders. The franchise’s projected $11 billion worldwide sales will put Argenx in 8th place in 2032, ahead of Merck & Co. and Bristol Myers Squibb.
Fifth placed Vertex Pharmaceuticals is the only other biotech within the top ten. Once-daily cystic fibrosis drug Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) is forecast to reach $9 billion by 2032, but predecessor Trikafta (elexacaftor/tezacaftor/ivacaftor), taken twice-daily, will remain among the front-runners with over $4.5 billion in sales.
Figure 3: WW Orphan Prescription Drug Sales in 2032: Top 10 Companies
Merck & Co. is the only top ten company expected to match Argenx’ anticipated 20%-plus compound annual orphan sales growth between 2025 and 2032, however – thanks to pulmonary hypertension drug Winrevair (sotatercept), projected to top $6.5 billion in 2032 sales. Next-fastest in growth terms is Sanofi, buoyed to 10.5% forecast growth by a half dozen orphan products within the top 100. The highest-ranked Sanofi drug, at 19th, is coagulation factor VIII replacement therapy Altuviio, forecast to top $3 billion by 2032.
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Figure 4: Worldwide Orphan Drug Sales (2025/2032): Top 20 Companies & Total Market
WW Orphan Sales ($bn)
Percentage of sales coming from orphan drugs
Company
2025
2032
CAGR
1
Johnson & Johnson
24.1
30.9
+5.2%
41%
40%
2
AstraZeneca
16.2
19.2
+3.4%
29%
26%
3
Novartis
12.8
19.0
+8.3%
24%
31%
4
Roche
13.9
18.7
+6.2%
23%
28%
5
Vertex Pharmaceuticals
11.6
17.5
+8.5%
97%
80%
6
Sanofi
10.2
16.8
+10.5%
21%
25%
7
Amgen
10.1
12.4
+4.1%
30%
32%
8
Argenx
4.1
11.2
+22.0%
100%
9
Merck & Co
3.5
9.9
+23.0%
6%
18%
10
Bristol Myers Squibb
10.7
9.7
-2.1%
Orphan sales at 10th-placed Bristol Myers Squibb will shrink over the next six years as competition finally punctures myeloma drug Revlimid’s patent and orphan exclusivity defences. Bristol’s newer orphans Reblozyl for beta thalassemia-associated anemia and CAR-T cell therapy Breyanzi for lymphoma are each anticipated to sell over $3 billion in 2032, but won’t fully fill the gap.
