When it comes to the most expensive medicines in the world, pharmaceutical companies point to the costly process of researching and developing new treatments, especially for uncommon diseases that represent a small market for the drug. In order to recoup costs, companies set prices that are well beyond the finances of most people, particularly for those already struggling with severe illnesses. In some of these cases, the drug is the only path to achieving any sort of progress or relief against the ailment and must be taken even if the medication costs the same as a small condo or a Lamborghini Aventador.
All of the medications on this list are for rare health disorders that, genetically, affect a very small portion of the population but often result in severe symptoms and problems.
The majority of the most expensive drugs on earth are enzyme replacement therapies that ease the symptoms of a disease. This prevents the body from dealing with sugars and proteins correctly. Other expensive medications include a remedy for a rare Cystic Fibrosis mutation, a tumor-shrinking drug and a substance that mimics that behavior of antibodies. These medications represent some of the most advanced medical practices, created through research at the genetic and molecular level in an attempt to recreate vital, natural processes in the human body.
11. Aldurazyme – $200,000 per year
Aldurazyme is prescribed to patients with Hurler Syndrome, a rare inherited disease with just under 600 diagnoses worldwide. This medication acts as a replacement therapy for enzymes that patients are unable to produce for themselves.
Without it, molecules of sugar accumulate within the body, including joints, mucus membranes, the heart, liver and other major organs. Aldurazyme is taken intravenously at a cost of about $200,000 per year. Unfortunately, even with this treatment, those who suffer from Hurler Syndrome still endure symptoms such as claw hand, stunted growth physically and intellectually, deafness and heart valve problems.
10. Cerezyme – $200,000 per year
Made by a recombinant DNA process that uses hamster ovaries, Cerezyme prevents fat from building up in the lungs, liver, bone marrow, spleen and the brain. This medication is another enzyme replacement therapy for a rare disease that causes interruptions to vital internal processes.
Striking between 1 in 50,000 to 100,000 people, Gaucher’s disease occurs in three different types, with type 2 and 3 causing potentially fatal problems in the nervous system and brain. Type 1 results in symptoms that range in severity, including anemia, easy bruising, brittle bones and lung disease.
9. Fabrazyme – $200,000 per year
Delivered intravenously every two weeks, Fabrazyme helps treat Fabry disease. This disease is an uncommon genetic disorder that prevents a certain type of fat from metabolizing, resulting in the disruption of a variety of biological systems. Symptoms include cloudy eyes, ringing in the ears, pain in the extremities and can result in heart attack, stroke and severe kidney damage.
About 1 in 40,000 to 60,000 men fall victim to the disease while women are much less likely to contract the ailment. This medication is noted for a high rate of severe reactions, sometimes requiring taking other drugs to mitigate the negative side effects.
8. Arcalyst – $250,000 per year
Arcalyst treats inflammatory problems in the body that are caused by rare genetic disorders, specifically, familial cold autoinflammatory syndrome – a disease that occurs in one in a million humans – and Muckle-Wells syndrome. Both these diseases cause the increased production of a protein called cryopyrin, which triggers an unnecessarily strong inflammatory response, especially in cold weather conditions.
7. Myozyme – $300,000 per year
Pompe disease is an inherited syndrome that prevents the normal processing of glycogen into glucose, a simple sugar and energy source for cells. Myozyme helps the body with this vital task, preventing a toxic buildup of glycogen throughout the body.
The medication is only available through special medical programs that ensure severe allergic reactions don’t take place during and up to three hours after the procedure.
6. Kalydeco – $307,000 per year
Kalydeco is a medication developed to combat the effects of cystic fibrosis among patients with a very specific genetic mutation in which the drugs becomes effective. This medication works to fix a defective protein that prevents fluids and salt from flowing in and out of cells properly.
5. Cinryze – $350,000 per year
Hereditary angioedema strikes from 1 in 10,000 to 50,000 members of the population, resulting in extreme swelling that can lead to life-threatening issues. Cinryze is prescribed to deal with the problems that occurs due to malfunctioning inhibitor proteins that cause the body to engage in swelling. Despite the fact that this disease is passed down in families, up to 20 percent of cases may happen due to a random mutation as a child is conceived. When angioedema attacks, intense abdominal pain, vomiting and nausea takes place, also potentially resulting in swelling of the airways to the point of asphyxiation.
4. Folotyn – $360,000 per year
Folotyn injections combat T-cell lymphoma after other courses of treatment fail, or in the event of a relapse of the cancer. However, the controversy behind this medicine is the fact that it hasn’t been shown to actually increase lifespan, despite the reduction of tumors.
3. Naglazyme – $365,000 per year
Naglazyme fights the effects of Maroteaux-Lamy syndrome, which is another rare, inherited condition that results in damage to the body due to the inability to properly process nutrients.
Sugars and proteins remain left behind, gathering in the eyes, skin, teeth, nervous system, respiratory system, liver and many other places in the body, resulting in significant damage.
Naglazyme aids patients by acting as a replacement for the malfunctioning enzymes. The meds are injected intravenously in a four-hour infusion. The treatment can result in easier walking and stair-climbing, even if it doesn’t cure the disease.
2. Elaprase $375,000 per year
About 2,000 people around the world suffer from Hunter syndrome, which is typically transmitted genetically to males due to a problem with the X chromosome. Along with many of the most expensive cures on this list, Elaprase is an enzyme replacement medication given intravenously. It assists with the processing of complex sugars, reducing the damage the disease causes the body.
1. Soliris – $409,500 per year
Alexion Pharmaceuticals manufactures Soliris to combat rare blood disorders by slowing the destruction of red blood cells. For those with paroxysmal nocturnal hemoglobinuria, a genetic disorder that speeds the breakdown of blood cells, Soliris helps reduce symptoms such as blood clots, pain, difficulty breathing and headaches. It also helps the patient survive as long as possible.
This drug is designed to behave in the same way as a natural antibody in the immune system, binding only to specific proteins that regulate the lives of red blood cells.