A dose of this medicine to treat rare disease will cost you millions

Hemgenix has been declared an effective treatment for the genetic blood disease hemophilia 'B'
Nov 30, 2022

The single-pill cure for hemophilia, Hemgenix, just established a new record for the costliest medicine ever at a staggering Rs780.86 million per dosage.

Hemgenix, a novel gene therapy developed to treat adults with haemophilia B, a life-threatening genetic illness in which patients cannot make enough of a blood-clotting protein, was given the green light by the US Food and Drug Administration on Tuesday.

Hemgenix has made it possible for people with hemophilia B to be permanently cured with a single IV infusion, as opposed to the several injections that were previously necessary to maintain adequate amounts of the missing protein.

The only catch is that Hemgenix is the world’s most expensive medicine, with a single dosage costing at least Rs780.86m.

Dr Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said, “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 experiencing a high burden of disease associated with this form of haemophilia.”

The Institute for Clinical and Economic Review, a nonprofit research group, recently conducted a cost-effectiveness study and concluded that a reasonable price for Hemgenix would be between Rs238m and Rs240.79m.

The mentioned price bracket is still completely out of reach for the typical individual.

Existing medicines for haemophilia B are likewise exceedingly costly, and individuals with the illness live in continuous dread of bleeding that might be fatal.

Hence, many feel that Hemgenix has a chance to be a successful medicine despite its high price.

The cost of gene treatments has steadily increased over time.

In 2018, we discussed the world’s most costly medicine, Luxturna, a single-dose gene therapy treatment for congenital blindness.

The price tag came up at above Rs191m, which was supposedly cheap.

Hemgenix has surpassed Zynteglo, a medicine used to treat beta thalassemia that was previously the most expensive drug in the world, with its price tag of above Rs631m.

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