What is the most expensive drug

The most expensive drugs in the world - Spinraza and Ravicti on top

New, life-saving or life-prolonging drugs keep coming onto the market. In 2016 alone, 22 new therapies were approved by the US Food and Drug Administration (FDA), and in 2017 there are already 20 new drugs that have successfully passed the approval process in the USA.

The majority of the new therapies come from US biotech companies. The treatment of rare diseases in particular is becoming increasingly expensive, as the research and development costs leading to the launch of a new drug have now risen above the US $ 2.5 billion mark.

Such drugs (orphan drugs) are correspondingly expensive, which is reflected in prices of several US $ 100,000 per year. The top 10 most expensive drugs in the world:

10. Naglazyme from Biomarin, cost: $ 485,747 per year

Naglazyme from the listed US biotech company Biomarin was developed and approved in 2005 for the treatment of patients with the rare disease mucopolysaccharidosis VI (MPS VI or Maroteaux-Lamy syndrome).

Naglazyme counteracts the deficiency of an enzyme (N-acetylgalactosamine-4-sulfatase). Only a few dozen people are diagnosed and treated with this therapy each year, and the cost of the drug adds up to over US $ 485,000 per year.

9. Idelvion from CSL Behring, cost: US $ 500,000 per year

At a cost of US $ 500,000, Idelvion from CSL Behring is also one of the top 10 most expensive drugs in the world. Idelvion is used to treat haemophilia B, also known as Christmas disease, which is caused by a congenital factor IX deficiency. The aim is to stop bleeding caused by the factor IX deficiency.

8. Alprolix from Bioverativ, cost: US $ 503,880 per year

The preparation Alprolix is ​​the only recombinant factor IX-FC fusion protein for the treatment of haemophilia B in the European Union (EU), which is supposed to offer extended protection against bleeding with just a few doses.

The preparation is used in particular for the prophylaxis of people with haemophilia B. The drug comes from the US biotech company Bioverativ, a venture by Biogen and Sobi, which has only recently been listed on the Nasdaq stock exchange.

7. Soliris from Alexion Pharmaceuticals, cost: $ 542,640 per year

Soliris from the US biotech company Alexion is also one of the most expensive drugs in the world, with annual costs of almost US $ 550,000. Soliris is a monoclonal antibody that is used to treat paroxysmal nocturnal hemoglobinuria (PNH) - a rare but life-threatening disease of the blood-forming stem cells.

6. Actimmune from Horizon Pharma, costs $ 572,292 per year

Actimmune (interferon gamma-1b) is a biological protein from Ireland-based biotech company Horizon Pharma.

This preparation is used to treat malignant osteopetrosis and chronic granulomatous disease (CGD), a genetic defect. Hopes that Actimmune could also be used to treat Friedreich's ataxia (disease of the central nervous system) have unfortunately not come true. Nonetheless, Actimmune treatment costs more than $ 572,000 annually.

5. Recordati Carbaglu, cost: $ 585,408 per year

Recordati's drug Carbaglu helps regulate NAGS deficiency in the body. N-acetylglutamate synthase deficiency (NAGS) is a genetic disorder that limits or blocks the body's ability to break down excess ammonia.

A high level of ammonia in the blood can lead to brain damage or even death. Since only a few people are affected by this genetic defect, the treatment is relatively expensive at over US $ 585,000 per year.

4. Lumizyme from Sanofi-Genzyme, cost: $ 626,400 per year

Lumizyme (alglucosidase alfa) is a drug from Sanofi-Genzyme and replaces the enzyme acid alfa glucosidase (GAA). This enzyme is absent in people with Pompe disease. Pompe disease occurs once in 40,000 to 300,000 births in a year and leads to weakness in the muscles of the internal and skeletal muscles, including weak breathing, which can also lead to death.

3. Brineura from Biomarin, costing $ 702,000 per year

Brineura (cerliponase alfa), also developed by the US biotech company Biomarin, which is one of the most innovative companies in the world, is new in the top 10 of the world's most expensive drugs. Brineura only received approval in the USA in April 2017 and is intended for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2).

This is a form of Batten's disease, a metabolic disease. With 20 diagnoses per year, CLN2 is one of the ultra-close diseases; the treatment is very expensive at US $ 702,000 per year.

2. Biogen's Spinraza, cost: US $ 750,000 per year

Spinraza (Nusinersen) was also only recently approved and is the world's first and only drug for the treatment of spinal muscular atrophy (SMA). The antisense oligonucleotide was developed by the US biotech companies Ionis Pharmaceuticals and Biogen and, with an annual cost of US $ 750,000 in the first year, is one of the most expensive drugs in the world. However, the treatment costs are halved after about a year.

1. Ravicti from Horizon Pharma, cost: $ 793,632 per year

At the top of the world's most expensive drugs is Ravicti, which is also sold by Horizon Pharma, based in Dublin / Ireland.

The drug was developed by Hyperion to treat urea cycle disorders. Horizon bought the company for $ 1.1 billion in 2015, strengthening its drug portfolio for rare diseases. With an annual cost of almost US $ 800,000, Ravicti is currently the most expensive drug in the world.

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