World’s costliest drug worth $3.5 million approved in US

Last week the United States Food and Drug Administration approved a new drug which is the most expensive one in the world and costs $3.5 million or more than Rs.28 crores per treatment.

The new drug Hemgenix is manufactured by CSL Behring and it is a gene therapy that treats haemophilia B – a genetic bleeding disorder in which patients are unable to produce a protein required to create blood clots.

This disorder affects 1 in 40,000 people in the world, most of whom are men.

The typical treatment for this disorder is routine intravenous infusions that help to maintain sufficient levels of the missing or deficient clotting factor. In the case of this new gene treatment, there is one-time IV infusion.

In a statement, Dr. Peter Marks, Director of the FDA’s Center for Biologics Evaluation and Research, described the therapy as representing an “important progress in the development of innovative therapies for those experiencing a high burden of disease associated with this form of haemophilia.”

Earlier, the most expensive drug in the US was Zolgensma which treats spinal muscular atrophy and is priced at $2.1 million for a course of treatment.