A costly new medication for a rare blood clotting condition has just been approved by the Federal Drug Administration (FDA) in the United States, according to a report by Science Alert.

It will be the most expensive medication in the world, costing $3.5 million for each dose.

(Photo : ERIC PIERMONT/AFP via Getty Images)
A laboratory staff works at Genethon, a non-profit gene therapy R&D organization in Evry on May 10, 2022. – Genethon, a non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon) is dedicated to the conception, clinic and preclinic development of drugs of genic therapy for rare diseases.

“Fair” Price

Although the price seems outrageous initially, a recent examination of the drug’s cost-effectiveness reveals that it is, at least in the US, a somewhat “fair” price for the treatment’s results.

The drug, Hemgenix, is a gene therapy treatment for hemophilia B, a rare genetic disorder that results in impaired blood clotting. The most dangerous signs include periods of difficult-to-stop spontaneous and recurrent bleeding.

Men are more likely than women to have hemophilia B, and although exact numbers are difficult to come by, estimations indicate that about 8,000 men in the US have this chronic illness.

Despite its high lifetime treatment costs, the main medication now used to treat hemophilia B in the US provides patients with the necessary clotting factor.

Patients with severe symptoms require a regular, costly treatment plan, one whose efficacy may gradually deteriorate over time, as per Science Alert.

The adult lifetime cost for each patient with moderate to severe hemophilia B is currently estimated by researchers to be between US$21 and $23 million.

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Hemgenix is a one-time intravenous medication administered in a single dose at a significantly reduced cost. A viral-based vector is used to deliver the product into the body and is designed to transfer DNA to aim at cells in the liver.

The instructions for a clotting protein called Factor IX are subsequently distributed by cells through the replication of this genetic material.

The effectiveness and safety of Hemgenix have thus far been examined in two studies. Increased Factor IX activity levels were discovered in one study among 54 participants with severe or moderately severe hemophilia B, negating the necessity for standard replacement medications currently offered to patients.

Patients’ rates of uncontrolled bleeding decreased by almost 50% in contrast to their baseline rates after undergoing gene therapy.

The FDA maintains a special designation for dangerous but uncommon diseases like hemophilia B to encourage medical research. For instance, Hemgenix is categorized as an “orphan drug” since it only has the potential to help a tiny number of patients.

This certification grants CSL Behring, the maker of Hemgenix, seven years of exclusive market access in the US.

The application of gene therapy is currently being evaluated by the European Medicines Agency and its drug regulatory counterparts in the UK and Australia.

Another “one shot” type of gene therapy for treating spinal muscular atrophy previously held the record for the most expensive medication. It was priced at $2 million per course.

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Written by Jace Dela Cruz

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