What is Hemgenix, world’s most expensive drug approved by FDA? How does it work?

Would you pay Rs 28.6 crore for a drug?

That’s how much the drug Hemgenix, recently approved by the US Food and Drug Administration to treat haemophilia, and made by Australian drugmaker CSL, costs per dose.

Let’s take a closer look at Hemgenix:

But first, let’s briefly examine haemophilia.

As per CDC, haemophilia is usually an inherited bleeding disorder in which the blood does not clot properly.

The disease primarily affects men and is caused by a gene mutation.

Blood contains proteins known as clotting factors that help stop bleeding.

However, those with haemophilia have low levels of either Factor VIII (8) or Factor IX (9).

A person’s severity of haemophilia is determined by the levels of Factor in their blood.

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The lower the amount, the more likely that bleeding could lead to serious health issues.

Haemophilia can result in:

Bleeding within joints that can lead to chronic joint disease and pain

Bleeding in the head and sometimes in the brain which can cause long term problems, such as seizures and paralysis

Death can occur if the bleeding cannot be stopped or if it occurs in a vital organ such as the brain.

What is Hemgenix?

Hemgenix is a one-off infusion which treats haemophilia B, a rare genetic blood clotting disorder.

Around one in 40,000 people is affected by haemophilia B – the condition caused by a gene mutation that hampers the body's ability to make Factor IX, as per Reuters.

As per Inshorts, Hemgenix works by delivering a gene that can produce the missing clotting factors into the liver, where it starts working to produce the Factor IX protein.

A study of Hemgenix found a single dose reduces the number of bleeding events over a year by 54 per cent, as per Hindustan Times.

This long-term solution is a contrast to current treatments from Biogen, Pfizer and others that require regular infusions.

The company’s CSL Behring, will begin commercialising the therapy under the brand Hemgenix immediately in the United States.

The drug will be manufactured in Lexington, Massachusetts, by uniQure NV, as per Hindustan Times.

Cowen analyst Joseph Thome said in a note Hemgenix's price is higher than the brokerage's estimated $1.9 million, but its consultants have been optimistic about the therapy's uptake.

Biotech investor Brad Loncar told the newspaper, “While the price is a little higher than expected, I do think it has a chance of being successful because 1) existing drugs are also very expensive and 2) haemophilia patients constantly live in fear of bleeds. A gene therapy product will be appealing to some.”

To be fair, the list price is not necessarily what the patients pay for the drug.

"We are confident this price point will generate significant cost savings for the overall healthcare system and significantly lower the economic burden of haemophilia B," the company said.

“Today’s approval provides a new treatment option for patients with haemophilia B and represents important progress in the development of innovative therapies,” FDA’s Dr Peter Marks was quoted as saying by Global News.

The FDA earlier cleared gene therapies Zynteglo and Skysona from Bluebird bio in August and September, respectively. While Zynteglo was priced at $2.8 million, Skysona had a wholesale cost of $3.0 million.

As per Global News, European regulators approved a similar gene therapy for haemophilia A. That drug, from drugmaker BioMarin, is still under review at the FDA.

With inputs from agencies

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