1. Glybera: $1.21 million wholesale cost per year.
We knew that one day we would see the world’s first seven-figure drug in terms of cost. Based on an approval in the EU last year for UniQure (NASDAQ:QURE), we now have one. Glybera is a gene therapy that helps restore LPL enzyme activity, which is critical to removing fat-carrying chylomicron particles in the intestines following a fat-containing meal. Specifically, it treats an extremely rare condition known as familial lipoprotein lipase deficiency, which affects only one in 1 million people — thus Glybera’s market potential is only around 150 to 200 people in the EU. Primary care physicians administer the drug in a one-time series of up to 60 intramuscular injections in a patients’ legs.When you consider its one-and-done dosing and extremely small patient pool, it’s slightly less shocking that UniQure settled on a 1.1 million euro price tag for Glybera.
2. Soliris: $700,000 wholesale cost per year.
Alexion Pharmaceuticals’ (NASDAQ:ALXN) Soliris has actually been the most expensive drug in the world for years, so seeing it fall out of the top spot may come as a surprise to many. Within the U.S. Soliris had a wholesale cost of nearly $537,000 last year, but the Patented Medicines Price Review Board notes that Soliris’ annual cost in Canada can be as high as $700,000 per patient. Soliris has two current indications: as a treatment for paroxysmal nocturnal hemoglobinuria (PNH), and as a treatment for atypical hemolytic uremic syndrome. Both are very rare indications with no current competition, thus the ability of Alexion to command such a high price tag for Soliris. More importantly, patients with PNH often live for 10 to 15 years following their diagnosis, so despite a small patient population, Alexion has a source of long-term recurring revenue with Soliris. After delivering $2.2 billion in revenue in 2014, Alexion is projected by Wall Street to more than double to $5 billion in 2018, mostly on the heels of Soliris.
3. Naglazyme: $485,747 wholesale cost per year (based on 2014 pricing).
Based on data provided by FiercePharma last year, BioMarin Pharmaceutical’s (NASDAQ:BMRN) Naglazyme will handily take the third spot, with an annual cost approaching a half-million dollars. I looked far and wide for an update on pricing for Naglazyme in 2015, but not a trace of an update was to be found. That’s probably because the drug is only prescribed to a few dozen patients on a per year basis, according to EvaluatePharma. Naglazyme is an enzyme replacement therapy designed to treat a disease known as mucopolysaccharidosis type VI, which is also known as Maroteaux-Lamy syndrome. This is a progressive disease that can cause organ enlargement and skeletal abnormalities, and it often leads to shortened life expectancies for those diagnosed.
4. Vimizim: $380,000 wholesale cost per year.
BioMarin Pharmaceutical, a rare disease specialist, also brings us the fourth-most expensive drug in the world with Vimizim. Vimizim, with its $380,000 per year cost, is an enzyme replacement therapy that’s given as a weekly infusion to treat Morquio A syndrome, a disease characterized by the body’s inability to break down long-chain sugar molecules. An estimated 800 people have Morquio A in the United States, and Vimizim’s developed world market is believed to be about 3,000 people. Analysts predict that despite its relatively small market potential, Vimizm could generate up to a half-billion in sales annually at its peak for BioMarin.
5. Elaprase: $375,000 wholesale cost per year (based on last update in 2010).
Lastly, the spot as fifth-most expensive drug in the world goes to Shire’s (NASDAQ:SHPG) Elaprase, an enzyme replacement therapy designed to treat patients with mucopolysaccharidosis II, or Hunter syndrome. You’ll note the last conclusive pricing data on Elaprase comes to us from 2010 when it ran $375,000 per year on a wholesale level, but there’s little reason to believe its price has fallen since that time with no added competition to the Hunter syndrome indication. Patients with Hunter syndrome lack an important enzyme (iduronate-2-sulfatase) that helps with the removal of long-chain sugar molecules. If these glycosaminoglycans aren’t removed from a patient’s body, it can lead to progressive organ decline, specifically of the heart, lungs, liver, and spleen. Incidence of the disease is fairly low, with the EU reported one case per every 140,000-156,000 births. With little standing in the way of higher prescription drug prices, annual costs in the high six-digit range could soon become a norm in the orphan disease space.