The uncommon genetic bleeding condition treatment developed by Pfizer was cleared by the Food and Drug Administration on Friday, marking the company’s first clearance for gene therapy in the United States.

The medication, which will be sold under the brand name Beqvez, was approved by the agency for use in people with moderate to severe haemophilia B who satisfy specific criteria.

This quarter, the therapy will be prescribed to qualified patients, according to a Pfizer representative. According to the spokesman, the medicine costs a whopping $3.5 million before insurance and other discounts, making it one of the priciest in the United States.

An advocacy organisation believes that the ailment, which mostly affects men, affects over 7,000 Americans.

The illness is brought on by low concentrations of a certain protein that aids in the formation of blood clots to seal wounds and stop bleeding. Patients with haemophilia B bruise more readily, bleed more often, and experience longer-lasting bleeding when that protein, known as factor IX, is absent.

Beqvez is a one-time medication that helps people stop and manage bleeding as well as create factor IX on their own. The medication outperformed the frequently laborious conventional therapy for haemophilia B, which entails injecting the protein into the veins many times a week or once a month, in a late-stage experiment.

“Many people with hemophilia B struggle with the commitment and lifestyle disruption of regular [factor IX] infusions, as well as spontaneous bleeding episodes, which can lead to painful joint damage and mobility issues,” said Adam Cuker, director of Penn Medicine’s Comprehensive and Hemophilia Thrombosis Program, in a Pfizer release on Friday.

“Pfizer’s medication “has the potential to be revolutionary for suitable individuals by lowering the long-term burden of both medical and therapy,” Cuker continued.

For Pfizer, which is attempting to recover its footing after the sharp collapse of its Covid business last year, the clearance marks a significant step. To help turn around its business, the corporation is placing a large wager on cancer medications and therapies for other diseases.

One of the many businesses investing in the quickly expanding field of gene and cell therapies is Pfizer. These are expensive, one-time medicines that target a patient’s genetic source or cell in an attempt to cure or drastically change the course of a disease. Certain medical professionals anticipate that these treatments will take the place of people’s long-term, conventional medications to address chronic diseases/ 

In 2014, Pfizer acquired the manufacturing and commercialization rights of Beqvez from Spark Therapeutics.

A business representative told CNBC that the company is providing payers with a warranty programme to cover patients who take Beqvez. According to the press announcement, Pfizer anticipates that the programme will provide “financial protection by insuring against the risk of efficacy failure”.

The gene therapy will face competition from CSL Behring’s Hemgenix, an Australian-based medicine that is comparable and was approved by the FDA in 2022 for haemophilia B. Before insurance and other discounts, such medication’s list price in the United States is comparable at $3.5 million.

Notably, some medical professionals have claimed that the adoption of Hemgenix and another authorised gene treatment for the more prevalent haemophilia A has been hampered by high prices and logistical challenges, among other reasons.

In order to treat haemophilia A and B, Pfizer is also requesting FDA clearance for marstacimab, an investigational antibody. Additionally, the business is working on a gene treatment for Duchenne muscular dystrophy, a hereditary condition that gradually weakens the muscles.

(Adapted from CNB.com)