US has approves world's most expensive drug at a cost of $4.25 million

The US Food and Drug Administration has approved Lenmeldy, a gene therapy drug for an ultra-rare childhood disease. A one-time treatment of Lenmeldy will cost $4.25 million, making it the most expensive drug in the world.

It is the first gene therapy for metachromatic leukodystrophy, a rare and fatal disease called metachromatic leukodystrophy with early onset, affecting predominantly children under the age of seven.

It is a fatal disease in which infants sometimes begin to lose the ability to walk and talk. Orchard Therapeutics, which makes Lenmeldy, said the drug's price "reflects its clinical, economic, and social value".The disease affects about one in 100,000 newborns and fewer than 40 children a year in the US, the statement said.

Orchard Therapeutics is developing gene therapies that aim to correct the underlying genetic defects that cause inherited diseases. The company was recently acquired by Japanese drug maker Kyowa Kirin Co.

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