The orphan drug sector has long been clearing growth in the larger pharmaceutical market with booming sales. But, thanks to blockbuster meds across wider disease areas and other challenges, the lucrative market may soon slow down for the first time in years.
According to Evaluate’s 2024 report, “Orphan Drugs are Losing Their Sparkle,” the category is set to pick up $185 billion this year and some $270 billion by 2028.
Yet, sales growth is notably shrinking. The meds averaged an almost 11% sales increase in the decade ending in 2023 but will "barely hit double digits" through 2030.
Several factors come into play to dull orphan drugs' shine, such as the “blazing return of big drugs for big diseases,” according to Evaluate. While the obesity market is currently the hottest ticket, blockbusters across disease areas such as neuroscience, immunology and oncology also stand to overtake orphan drug’s slice of the pie.
Plus, there are pricing pressures stemming from the high price tag on orphan drugs. Payers are “more discerning” as of late and aren’t as keen to cover the steep prices, favoring more widespread disease areas instead, according to Evaluate.
Still, the sales slowdown is “relative,” head of thought leadership at Evaluate Daniel Chancellor said in a press release. “As the overall prescription drug market continues to grow, so will that for rare diseases.”
2028's top orphan drugs
According to the report, the top 10 most valuable orphan drugs will together collect more than $57 billion in 2028. That top 10 list has had a bit of a shakeup since Evaluate’s last ranking in 2023, largely due to increasing competition and the Inflation Reduction Act.
Johnson & Johnson’s multiple myeloma blockbuster Darzalex, Vertex’s cystic fibrosis med Trikafta and Roche’s hemophilia A therapy Hemlibra all kept their top three slots on Evaluate’s list of the 10 top sellers by 2028 in worldwide sales with predicted respective revenues of $17 billion, $8.7 billion and $6.2 billion.
However, AbbVie’s popular cancer med Imbruvica has fallen out of the top 10 thanks to Beigene’s rival BTK inhibitor Brukinsa and AstraZeneca’s Calquence. While Calquence is slated for $4 billion in 2028 sales and Brukinsa should pick up $3.8 billion, AZ’s offering fell from fifth place to seventh over the past year.
Argenx’s Vyvgart also slipped, dropping to 14th place with a forecast $3 billion in 2028. The generalized myasthenia gravis drug is challenged by growing competition such as UCB’s two new treatment options, Zilbrysq and Rystiggo, plus upcoming threats from Johnson & Johnson’s nipocalimab.
Despite label expansions last year, Roche’s spinal muscular atrophy therapy Evrysdi had a similar story with competition from Biogen's Spinraza and Novartis' Zolgensma.
Meanwhile, new entrants to the top 10 list included Incyte and Novartis’ JAK inhibitor Jakafi, which placed sixth with a 2028 sales expectation of $4.2 billion, and J&J’s Carvykti. The latter is the only CAR-T therapy to appear on the top 10 list with $4.4 billion in predicted 2028 sales.
As for the wider 2028 picture, Amgen swiped AbbVie’s spot on the list of the top 10 companies with largest orphan drug sales. That can be attributed to Amgen’s $27.8 billion acquisition of rare disease drugmaker Horizon Therapeutics, which brings orphan drugs Tepezza, Krystexxa and Uplizna into the fold.
The top spots in that ranking go to J&J, Roche and AZ, with J&J taking No. 1 in a landslide. The company is expected to bring in over $30 billion in 2028 orphan drug sales, while Roche at No. 2 looks to take home $16.8 billion.