The highest-priced drugs have two things in common: they all treat rare diseases, and they all cost hundreds of thousands of dollars per year. A report posted on the Motley Fool website lists the world’s seven most-expensive drugs and the companies that profit from them.
Glybera (alipogen tiparvovec) is a gene therapy for adults with familial lipoprotein lipase deficiency, a rare genetic disorder that causes fat to accumulate in the blood. The drug, which won European approval in October 2012, costs more than $1 million per year. The FDA requested additional clinical trials before it would review Glybera for approval, but the drug’s developer––Dutch pharma company Uniqure––decided not to move forward with those studies.
Ravicti (glycerol phenylbutyrate), a nitrogen-binding agent, is used to treat urea cycle disorders (UCDs)––genetic diseases that prevent the body from eliminating ammonia. The resulting buildup of the toxic substance can lead to brain damage and death. Horizon Pharma sells Ravicti at an annual cost of nearly $794,000.
Spinraza (nusinersen, Biogen) received FDA approval in December 2016 for use in patients with spinal muscular atrophy. The drug costs $750,000 for the first year of treatment, but the price tag drops to $375,000 thereafter because fewer injections are required in subsequent years.
Lumizyme (alglucosidase alfa, Sanofi) replaces a missing or deficient enzyme in patients with Pompe disease, a genetic disorder caused by the buildup of glycogen in cells. The drug costs more than $626,000 annually.
Like Ravicti, Carbaglu (carglumic acid) is used to treat patients with UCDs. Unlike Ravicti, however, Carbaglu specifically targets N-acetylglutamate synthetase deficiency. The drug’s developer, Recordati Rare Diseases, has set an annual price of $585,000.
Horizon Pharma, the maker of Ravicti, also markets Actimmune (interferon gamma-1b), a biologically manufactured protein, which is used to treat two rare genetic disorders: chronic granulomatous disease (CGD) and severe malignant osteopetrosis (SMO). The drug is priced at more than $572,000 per year.
Soliris (eculizumab, Alexion Pharmaceuticals) also treats two rare diseases: paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). The annual cost is nearly $543,000.
As of 2015, more than 90 other prescription drugs carried annual price tags of more than $100,000, according to the Motley Fool article. Five of them cost more than $400,000 per year––just behind the drugs that made the Top Seven list.
Source: Motley Fool; April 18, 2017.