Top 5 Most Expensive Drugs
1. Glybera® (UniQure Inc.) - $1.21 million per year
Glybera is the first gene therapy drug approved in Europe but yet to be approved in the US. It helps treat lipoprotein lipase deficiency. People with this deficiency cannot break down fat, leading accumulation of fat in the blood.
2. Ravicti®/ (Horizon Pharma) - $793,632
This medication is for urea cycle disorders (UCDs) and accounted for $151 million in net sales in 2016. In UCDs, nitrogen accumulates leading to hyperammonemia which can cause irreversible brain damage.
3. Lumizyme® (Sanofi Genzyme) - $626,400
This therapy is used treat Pompe disease, a rare disease that affects 40,000 live births annually and about 5,000 to 10,000 people worldwide suffer from it. Pompe disease or glycogen storage disease type II is a metabolic disorder that damages the muscle and nerve cells.
4. Carbaglu® (Recordati) - $585,408
This drug is an oral treatment for patients with hyperammonemia due to NAGS deficiency.
5. Actimmune® (Horizon Pharma) - $572,292
This treatment is indicated for maglinant osteoporosis and chronic granulomatosis. This medication was original developed by InterMune and then was bought by Vidara Therapeutics in 2012. Then in 2014, Horizon Pharma bought it for $660 million.
Source: http://www.fiercepharma.com/marketing/world-s-most-pricey-drugs-from-a-1-2m-gene-therapy-to-a-450k-lymphoma-med