In recent years, orphan drugmakers have stormed the market, racking up serious sales in small patient pools and dodging much of the generic and me-too competition that large-market drugs often face. And their piece of the pie is only set to get bigger, a new report says.
Barring any significant payer protest, orphan drug sales will make up 19% of the total share of prescription drug sales by 2020, totaling $176 billion, EvaluatePharma predicts in its Orphan Drug Report 2014. And they'll grow at an annual rate of nearly 11% per year through the end of the decade, compared with about 4% for drugs treating larger populations.
Driving that rise will be a mix of older orphan-category stalwarts, brand-new entrants in the red-hot immunotherapy field, and yet-to-win-approval up-and-comers, EP expects. A little more on its forecast top 10 orphan sellers by 2020:
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Revlimid: Celgene's ($CELG) top dog may be shining now, with $4.28 billion in sales last year. But by 2020, that's set to nearly double, according to EP. The blood cancer drug will see its sales swell to hit $8.01 billion in 2020 to top the orphan drug market.
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Opdivo: Bristol-Myers Squibb's ($BMY) cancer prospect so far only has regulatory approval in Japan, but it has the attention of industry-watchers around the world. That's because analysts predict sales of the drug--which strips cancer cells of a cloaking mechanism that keep them hidden from the immune system--will skyrocket, with EP forecasting it'll hit the $6.03 billion yearly sales mark within the next 5+ years.
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Rituxan: Roche's ($RHHBY) megablockbuster is no newcomer to the orphan drug field; on the contrary, its U.S. patent is set to expire in 2018. But even with biosimilars makers on its tail, EP predicts the chronic lymphocytic leukemia drug can still churn out $5.69 billion in 2020 sales--a near-32% dip from its 2013 haul.
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Soliris: Alexion's ($ALXN) high prices--the drug was ranked the most expensive in the world last year--will help keep the revenues coming for its ultrarare-disease star, which treats paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. Its $536,629-per-year price tag will help it go from $1.55 billion last year to $5.51 billion by the time the end of the decade rolls around.
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VX-809 + ivacaftor: Vertex's ($VRTX) cystic fibrosis drug Kalydeco has already done well for itself in a small population, with the company reaching most eligible patients. But with a new combo filed Wednesday for U.S. and EU approval, it could potentially do a whole lot better: ISI Group analyst Mark Schoenebaum said in a recent note that more than 28,000 patients could be eligible for treatment with the cocktail, which EP thinks could reach $4.23 billion in 2020 sales.
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Keytruda: Alongside Bristol-Myers' nivolumab, Merck's ($MRK) Keytruda has been another closely watched drug in the PD-1 class, grabbing FDA approval in September. Its novel approach to attacking cancer, combined with a $150,000-per-year sticker price, should bring it to $3.88 billion in 2020 sales, EP predicts.
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Imbruvica: This Johnson & Johnson ($JNJ) med hit the ground running after collecting three FDA breakthrough designations along the regulatory pathway. The B-cell cancer med, which currently boasts indications in CLL, is set to generate $3.5 billion in 2020, EP figures.
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Gazyva: With Rituxan revenues predicted to decline over the next few years, Roche is ready with a successor. Its follow-up to the CLL giant, Gazyva, has already jumped into the mix, and EP sees it bringing in $2.53 billion in 2020 as Rituxan wanes.
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Obeticholic acid: Intercept's ($ICPT) Phase III prospect, which mimics human bile acid, is in development for primary biliary cirrhosis, nonalcoholic steatohepatitis and other liver and intestinal diseases. Assuming it grabs FDA approval, EP thinks it can rake in $2.52 billion by 2020.
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Elelyso: Pfizer's ($PFE) Elelyso is playing in a crowded field for Gaucher disease, battling competitors from Shire ($SHPG), Actelion ($ATLN) and Sanofi ($SNY) in the U.S.--and more may be on the way. But that won't stop the drug giant from taking its sales from $49 million last year to $2.48 billion in 2020, the way EP sees it.
- download EP's report
Special Report: Top 20 orphan drugs by 2018