What’s the most expensive medication in the world?

The American publishing and media company, Forbes, assembled a list of the world’s most expensive medications. They obtained the average medication price per patient per year by interviewing biotechnology industry experts and Wall Street analysts. According to Forbes, nine medications cost more than $200,000 per year. What are they?


#9 Laronidase (Aldurazyme®): $200,000 per year

This agent acts as enzyme replacement therapy in patients with mucopolysaccharidosis I (MPS I, Hurler syndrome). MPS I is a lysosome storage deficiency caused by a lack of the lysosomal hydrolase, alpha-L-iduronidase. This deficiency results in the accumulation of heparan sulfate and dermatan sulfate in the lysosome. The syndrome is characterized by skeletal abnormalities, hepatosplenomegaly and intellectual disability. It affects about 600 people in the world.


#8 Agalsidease β (Fabrazyme®): $200,000 per year

This drug is enzyme replacement for patient with Fabry disease. In this disease, a deficiency in another lysosomal hydrolase, alpha-galactosidase-A, causes globotriaosylceramide to accumulate within lysosomes. The manifestations of Fabry disease are nonspecific, but include cardiovascular and renal complications. Around 2,200 patients in the world are affected.


#7 Imiglucerase (Cerezyme®): $200,000 per year

Enzyme replacement in patients with Gaucher disease. The pathophysiology of this disease is similar to that of Fabry disease and results in the build-up of lipid-laden macrophages in the spleen, bone marrow and other organs/tissues. About 5,200 patients worldwide have the disease.


#6 Rilonacept (Arcalyst®): $250,000 per year

Rilonacept inhibits inflammation by binding IL-1β and preventing its interaction with cell surface receptors. It’s indicated in patient with familial cold autoinflammatory and Muckle-Wells syndrome. Patients with these syndromes carry a rare genetic mutation that ultimately results in a deficiency of cryopyrin, a protein that regulates IL-1β. An estimated 2,000 people worldwide have Muckle-Wells syndrome.


#5 Alglucosidase alfa (Myozyme®): $300,000 per year

This drug is replacement for patient with a deficiency or absence of acid alpha-glucosidase, which is known as Pompe’s disease. An insufficiency of this enzyme results in glycogen accumulation in the tissues, progressive muscle weakness and an enlarged heart. Only around 900 people in the world suffer from Pompe’s disease.


#4 C1 Inhibitor (Cinryze®): $350,000 per year

C1 inhibitor is an acute-phase reactant protein that inhibits the classical complement pathway as well as the intrinsic coagulation (contact) system. In patients with hereditary angioedema (HAE), C1 inhibitor is thought to suppress activation of the contact system and ultimately prevent bradykinin production. Around 6,000 people in the US have HAE.


#3 Galsulfase (Naglazyme®): $365,000 per year

Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome) is another lysosomal storage disorder. MPS VI is caused by a deficiency in N-acetylgalactosamine 4-sulfatase and patients affected by the syndrome are characterized by an enlarged head, thick lips and short stature. An estimated 1,100 people have MPS VI. Galsulfase is a recombinant form of N-acetylgalactosamine 4-sulfatase. It’s produced in Chinese hamster cells.


#2 Idursulfase (Elaprase®): $375,000 per year

Replaces iduronate 2-sulfatase in patients with MPS II (Hunter syndrome), which is yet another lysosomal storage disorder. The syndrome is characterize by cardiac, respiratory and mobility dysfunction and affects an estimated 2,000 patients worldwide.


And the most expensive drug in the world is….


#1 Eculizumab (Soliris®): 409,500 per year

This price is based on the treatment regimen for paroxysmal nocturnal hemoglobinuria (PNH). In the US, 8,000 people carry a diagnosis of PNH. Eculizumab is also FDA approved for the treatment of atypical hemolytic uremic syndrome (aHUS) and has recently been studied for preventing antibody-mediated rejection in highly sensitized kidney transplant recipients. Eculizumab is a monoclonal antibody that inhibits complement activity by prevents the formation of the membrane attack complex (MAC). MAC causes cell lysis and death by disrupting the phospholipid bilayer of target cells.


1.     http://www.forbes.com/2010/02/19/expensive-drugs-cost-business-healthcare-rare-diseases.html