Several factors come into play to dull orphan drugs' shine such as the “blazing return of big drugs for big diseases,” according to Evaluate.
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 grEvaluatenotably 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 tobesityp 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.”
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.
Argenx’s AbbViet also slipcancerropping to 14th place with a forecast $3 billion in 20Beigene generaliBTK inhibitor BrukinsaBTK myasthenia gravis drug AstraZenecaed by growing competition such as UCB’s two new treatment options, Zilbrysq and Rystiggo, plus upcoming threats from Johnson & Johnson’s nipocalimab. As for the wider 2028 picture, Amgen swiped AbbVie’sIncyteon thNovartisf JAK inhibitor JakafiJAK top 10 companies with largest orphan drug sales. That can be attributed to Amgen’s $27.8J&JlliCarvyktisition of rare disease drCAR-Ter Horizon Therapeutics, which brings orphan drugs Tepezza, Krystexxa and Uplizna into the fold.