Thursday, July 04, 2013

The 5 Most Expensive Drugs in the World

According to research conducted by Forbes, the five most expensive drugs in the world each cost upwards of $300,000 annually.


Developed by Alexion Pharmaceuticals (NASDAQ: ALXN  ) , Soliris is the most expensive drug in the world, at an average annual cost of $409,500. Soliris itself treats a rare blood disorder called paroxysmal nocturnal hemoglobinuria, in which patients' red blood cells break down faster than they're supposed to. The reason is a missing gene known as PIG-A, whose function is to allow proteins to stick to red blood cells and protect them from substances in the blood that would otherwise cause them to break down. Without this protein present, anemia and other serious complications can develop. There are only about 8,000 known cases of PNH in the United States.

Soliris added another indication in 2011 to treat atypical hemolytic uremic syndrome, or aHUS, a rare and life-threatening genetic disease that can adversely affect the kidneys, heart, and brain. It's estimated there are only 300 known cases in the United States.


Elaprase, which is made by Shire (NASDAQ: SHPG  ) , runs at a bargain-basement annual cost of "just" $375,000 when compared to Soliris. Elaprase is designed to improve walking function in patients with Hunter Syndrome, a disorder whereby an enzyme the body needs to break down complex molecules is missing or not functioning properly. Without this enzyme present or working properly, these molecules can build up in the brain and other tissues, causing permanent damage. There are an estimated 2,000 known cases worldwide.


Coming in third with an annual cost of $365,000 is BioMarin Pharmaceutical's  (NASDAQ: BMRN  ) Naglazyme. This drug is an enzyme replacement therapy for patients with mucopolysaccharidosis VI, also thankfully abbreviated for us as MPS VI. The disease afflicts approximately 1,100 people worldwide and is characterized by an enlargement of tissues and organs that can result in inflammation and permanent scarring.


Acquired by ViroPharma (NASDAQ: VPHM  ) when it purchased Lev Pharmaceuticals in 2008, Cinryze slides in as the fourth-most expensive annual drug, costing insurers and patients $350,000. Cinryze was developed to treat a condition known as hereditary angioedema, a rare genetic disorder characterized by recurrent bouts of severe swelling that can affect the face, limbs, abdomen, and airway. There are about 6,000 cases of HAE in the United States. 


Not to be forgotten as the fifth-most expensive drug in the world is Sanofi's  (NYSE: SNY  )  Myozyme, which can be yours for the low, low price of $300,000 per year. Myozyme is designed to treat Pompe disease, a disorder characterized by a mutation to the GAA enzyme that subsequently allows glycogen to build up in the body -- specifically affecting the heart and skeletal muscles. There are only 900 known cases of Pompe disease around the globe.


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