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FDA Approves Most Expensive Drug Ever, Priced At $3.5 Million-Per-Dose For Hemophilia

A $3.5 million (₹285,320,350.00 INR) one-time treatment for hemophilia has been approved by U.S. FDA on Tuesday. For adults with hemophilia B, a less common form of the genetic disorder which primarily affects men, the Food and Drug Administration has approved Hemgenix, an IV treatment for the disease [1].

Details About The Most Expensive Drug Ever

"Today's approval provides a new treatment option for patients with hemophilia B and represents important progress in the development of innovative therapies," said the FDA's Dr. Peter Marks [2].

Point 1: Hemgenix is the most expensive medicine in the world according to a study cited by the National Library of Medicine, easily topping Novartis' Zolgensma gene therapy for spinal muscular atrophy (SMA), which costs just over $2 million (163,310,022 INR per dose) and is also a single-dose treatment [3].

Point 2: According to the agency, patients should benefit from the treatment for years to come due to reduced bleeding and increased clotting.

Point 3: The Pennsylvania-based drugmaker CSL Behring announced the price tag shortly after FDA approval, claiming the drug would ultimately reduce health care costs by reducing bleeding incidents and clotting treatments.

Point 4: Most of the costs of the new treatment will be covered by insurers, not patients, including private plans and government programs [4].

What Is Haemophilia?

A person with haemophilia usually has an inherited bleeding disorder that can cause spontaneous bleeding as well as bleeding following an injury or surgical procedure. The blood contains several proteins called clotting factors that can prevent bleeding [5].

The two most common types of haemophilia are Haemophilia A (Classic Haemophilia), caused by a lack of or decrease in clotting factor VIII, and Haemophilia B (Christmas Disease), caused by a lack or decrease in clotting factor IX.

One in every 5,000 males is born with haemophilia A, which is four times as common as haemophilia B, and about half of those with haemophilia A are affected by the severe form [6].

It is rare for someone to develop haemophilia at a later stage in life. In this case, hemophilia tends to affect people in their middle age or elderly, as well as young women who have recently given birth or are in the later stages of pregnancy. Treatment is usually effective in resolving this condition.

When left untreated, haemophilia can cause bleeding that seeps into joints and internal organs, including the brain. Small cuts and bruises can cause life-threatening bleeding for people with haemophilia.

The prevalence of haemophilia in India is low, at 0.7 per 1,000 people.

Story first published: Thursday, November 24, 2022, 10:35 [IST]
Read more about: fda hemophilia treatment drug
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