Regulators have approved the world's most expensive medicine that costs $3.5 million per patient

The world's most expensive medicine has been approved by the US.

Hemgenix is the first gene therapy to treat adults with a genetic bleeding disorder caused by missing or insufficient levels of Factor IX.

One in 40,000 people were affected by the condition. It makes up 15% of all cases of the disease.

The number of bleeding events expected over a year was cut in half by the distribution of Hemgenix. It saved patients a lot of time and money by removing the need for most of them to get Factor IX.

Peter Marks is the director of the FDA's Center for Biologics Evaluation and Research.

Today's approval provides a new treatment option for patients with hemophilia B and represents important progress in the development of innovative therapies for those with this form of hemophilia.

The drug will cost $3.5 million per dose, making it the most expensive medicine in the world.

The price point will generate significant cost savings for the overall healthcare system and significantly lower the economic burden of hemophilia B by reducing annual bleed rates, reducing or eliminating prophylactic therapy and generating elevated FIX levels that last for years, according to a CSL spokesman.

The price is higher than the Institute for Clinical and Economic Review's recommendation.

Brad Loncar, the chief executive of Loncar Investments, said in an interview that he thought the treatment could be successful because existing drugs were expensive and patients were afraid of bleeding.

The list price for Hemgenix is more than double that of Zynteglo which was priced earlier this year.