5 Most Expensive Drugs in the World

In this article, we will take a look at the 5 most expensive drugs in the world. If you want to read our discussion on the pharmaceutical industry, you can go directly to the 20 Most Expensive Drugs in the World.

5. Myalept

Cost: $1.26 million

Company: Chiesi Farmaceutici

Myalept is given in combination with a proper diet for leptin deficiency in people with lipodystrophy, also known as fat redistribution. Lipodystrophy is a condition affecting how the body stores fat. The approval for Myalept was given on February 23, 2014. The high cost of Myalept is mainly due to the drug being the only approved treatment for leptin deficiency.

4. Zolgensma

Cost: $2.25 million

Company: Novartis

Zolgensma is a single-dose treatment designed to address the genetic origin of spinal muscular atrophy. It works by replacing the function of the absent or malfunctioning SMN1 gene, stopping the advancement of SMA. This gene therapy is specifically used for the treatment of SMA in children under the age of 2.

3. Zynteglo

Cost: $2.8 million

Company: Bluebird Bio

Zynteglo is a gene therapy that involves utilizing a patient’s hematopoietic stem cells. This therapy was granted approval on August 17, 2022. It is specifically used for treating beta-thalassemia in adults and pediatric patients who depend on regular red blood cell transfusions.

2. Skysona

Cost: $3 million

Company: Bluebird Bio

Skysona is a single-dose gene therapy used as a treatment for boys experiencing early and active cerebral adrenoleukodystrophy (CALD). CALD results from genetic mutations in the ABCD1 gene, triggering the long-chain fatty acid in the brain.

1. Hemgenix

Cost: $3.5 million

Company: CSL Behring

Hemgenix is used in treating moderate to severe hemophilia B in adults. This bleeding disorder results from a deficiency of factor IX, an important protein for blood clot formation to control bleeding. Hemgenix was recognized as an ‘orphan medicine’ on 21st March 2018.