Meet the world’s most expensive drug worth 3.5 million dollars a dose

As you all know, healthcare is one of the most important yet expensive aspects of our lives. Treatments of ailments like cancer, diabetes, hypertension, heart-related diseases come with hefty price tags.  

But guess what? The world’s most expensive drug doesn’t cure any of this. It’s a newly approved course of treatment for a rare and genetic blood clotting disease called haemophilia. Named Hemgenix, the medicine launched by CSL Behring costs a whooping 3.5 million US dollars (or INR 28.58 crores) that has recently been given a nod by the United States Federal Drug Administration, better known as FDA.  

Sources cite that the drug may appear to be super expensive at a glance, but in reality, is cost-effective, keeping in mind the overall amount of money it takes to treat a patient suffering from haemophilia.  

Okay, so how exactly will Hemgenix work? Well, in haemophilia (especially for stage B patients), the most serious symptoms are random and repeated bleeding episodes, that are often difficult to control. And this medicine will be effective in the sense that once consumed, it will conduct its own gene therapy and keep the blood tapped in, rather than oozing out. Simply put, the drug will be pushed into a human body by a viral-based vector, which is designed to deliver alternative DNA (to fill up the missing clotting factor) to target cells in the liver. Once the genetic information is replicated by the said cells, the clotting protein called Factor IX knows exactly what to do (perform blood clotting like usual). 

While haemophilia can occur in both males and females, it’s more common in men. As per estimates, in USA alone, around 8000 men is currently suffering from this lifelong disease. Statistics also suggest that lifetime expenditure for every patient suffering from haemophilia in USA stands at 21 to 23 million US dollars, while in UK it’s 10 million pounds. In contrast, a single dose of the one-time intravenous drug Hemgenix can be afforded at fraction of that price. 

So far, two trials have been conducted with Hemgenix, In the first, 54 participants were severe or moderate haemophilia B shown signs of improved health (the rate of uncontrolled bleeding dropped by over 50 percent in a span of a year), in turn reducing the need for regular treatments.  

However, just like any other heavy medicines, this too has its own set of side effects such as headaches, flu, elevated enzymes in the liver – all of which can be monitored by physicians, that too quite easily.  

Interestingly, experts aren’t yet certain whether this drug is a cure for haemophilia that the world has been looking for, or just a controlled medication. Regardless, the results so far have been positive and promising. However, it has also been said that Hemgenix may be an “orphan drug” meaning it may not treat everyone with the condition, but only a small number of patients. Currently, UK and Australia are two other nations to have shown interest in the drug. 

Earlier, the most expensive drug was another one-time gene therapy course of treatment for spinal muscular atrophy. It was priced at 2 million US dollars.