The market for orphan medicines – those intended for diseases so rare that the supply of medicines is low – is expected to generate US$ 185 billion (around R$ 929 billion) this year and reach the mark of US$ 270 billion (around R$ 1.4 trillion) by 2028. The data is from Evaluate Pharma, which places Janssen, Vertex and Roche as the leaders in the segment.
However, the study highlights that the profits recorded by other categories have been of more interest to the pharmaceutical industry than the returns achieved with niche medicines. In addition to the treatment of obesity, which has attracted attention from all over the world, other areas such as immunology, neuroscience and oncology have also been attracting focus from various laboratories.
The complexity of R&D and the high cost of orphan drugs have helped to shift investments towards research that will serve a wider audience.
Top 10 of orphan drug market in 2028
| Rk | Medicine | Manufacturer | Therapeutic class | Revenue US$/R$ (billion) |
| 1 | Darzalex | Janssen | Oncology | US$ 17 / R$ 88.32 |
| two | Trikafta | Vertex | Respiratory | US$ 8.75/ R$ 45.46 |
| 3 | Hemlibra | Roche and Chugai Pharmaceutical | Blood | US$ 6.2/ R$ 32.21 |
| 4 | Ultomiris | AstraZeneca | Immunomodulator | US$5.18/ R$26.91 |
| 5 | Carvykti | Janssen and Legend Biotech | Oncology | US$4.4/ R$22.86 |
| 6 | Jakafi | Incyte and Novartis | Oncology | US$4.2/ R$21.82 |
| 7 | Calquence | AstraZeneca | Oncology | US$4/ R$20.78 |
| 8 | Brukinsa | BeiGene | Oncology | US$ 3.8/ R$ 19.74 |
| 9 | Lynparza | AstraZeneca | Oncology | US$ 3.4/ R$ 17.66 |
| 10 | Venclexta | AbbVie | Oncology | US$ 3.3/ R$ 17.14 |
* Source: Evaluate Pharma
