Kallanum Bhagavathiyum
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The world’s most ‘expensive medicine’ has been approved; Read on…

The most costly medication in the world, a treatment to treat the uncommon genetic condition haemophilia B, was authorised by the Food and Drug Administration (FDA), a US regulatory authority, on November 22. The treatment known as Hemgenix, manufactured by the CSL Behring pharmaceutical business, is the most expensive medication in the world according to Bloombergreports. It uses gene therapy and costs $3.5 million each dosage. Clinical research has shown that Hemgenix can prevent the disease-related bleeding episodes in more than half of the individuals studied, potentially reducing healthcare expenses.

According to the Higher Institute of Health (ISS), haemophilia B affects one in 30,000 people in the general population and is a rare genetic illness brought on by a deficiency in a protein needed to control bleeding in the event of injury. Being a condition linked to the sex chromosome X, men are more likely to have symptoms than women, who are typically healthy carriers. In order to avoid bleeding or to stop it on demand, the missing clotting factor is typically administered as part of this disease’s therapy.

Functions 
Hemgenix, however, operates differently: it is an adenoviral vector given intravenously in a single dosage, carrying the gene for the deficient coagulation factor. Once the gene is in circulation, the liver expresses it, producing the protein required to stop bleeding episodes.

Tests
Two clinical studies involving 57 men with severe or moderately severe haemophilia B were conducted to assess the safety and efficacy of Hemgenix. In one of the studies, participants experienced higher levels of missing clotting factor activity, which reduced the need for preventive treatment and resulted in a 54% decrease in the rate of annual bleeding events.

Cost considerations
Hemgenix would cost 3.5 million dollars each dosage as a result of the new drug’s innovativeness, which comes at a hefty price in this situation.¬† Instead, according to a research conducted by the Institute for Clinical and Economic Review, a non-profit group concerned with the prices of medications, a reasonable price for Hemgenix would be between $2.93 million and $2.96 million per dosage.

 

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