This disreputable company is managing to screw the public so many different ways. Here is my article from November 20 about awful CSL-Sequirus products. Below is today’s article from STAT on its affiliate CSL-Behring’s new hemophilia drug, based on a trial in 54 people.

Sporting a $3.5M price tag, CSL and uniQure's hemophilia B gene therapy crosses FDA finish line

After an FDA endorsement Tuesday, CSL Behring and uniQure's hemophilia B gene therapy Hemgenix debuts not only as the first gene therapy approved for the rare disorder, but as the new most expensive drug in the world. The drug will sport an eye-popping price tag of $3.5 million per dose.

Hemgenix is approved for patients who currently use factor IX prophylaxis therapy or have serious hemorrhage or spontaneous bleeding episodes. CSL Behring will commercialize the gene therapy after snapping up global rights from uniQure for $450 million up front back in 2020.

Despite the high price, the drug will generate “significant cost savings for the overall healthcare system” and “significantly lower the economic burden of hemophilia B by reducing annual bleed rates, reducing or eliminating prophylactic therapy and generating elevated [factor] IX levels that last for years," CSL Behring said in an emailed statement to Fierce Pharma.

Patients with moderate to severe hemophilia B can cost healthcare systems more than $20 million over their lifetimes, the company said. [Most of these high cost-generating hemophilia patients were knocked off in the 1980s by HIV-contaminated blood products, saving insurers billions. “It is estimated that about 50% of all those with hemophilia became infected, including around 90% of those with severe hemophilia. Thousands of people with hemophilia developed AIDS and died from this disease.”—Nass] CSL Behring also noted that the price does not reflect anticipated discounts that the company will provide for the therapy, including value-based agreements with commercial payers.

But the cost is still higher than the recommended price from Boston’s Institute for Clinical and Economic Review (ICER). Early this month, ICER said Hemgenix would be fairly priced at upwards of $2.9 million. In the report, ICER supported a multimillion-dollar price tag because of “health gains and the potential for substantial cost offsets due to elimination of the need for very expensive prophylactic treatment."

Hemophilia B is caused by a gene defect that causes patients to not produce enough factor IX, a protein that helps form blood clots and is mainly produced by the liver. Historically, patients had to turn to prophylactic infusions of factor IX replacement therapy to supplement low levels of the blood-clotting factor. Since the disorder is a life-long affliction, strict infusion schedules can be now replaced by one injection of Hemgenix, which allows patients to continuously produce their own factor IX.

The FDA granted the approval based on results of the HOPE-B trial, the largest gene therapy trial in the disorder to date with 54 participants. Seven to 18 months after infusion, patients' mean annual bleeding rates fell by 54% compared with their six-month lead-in period. A whopping 94% of patients stopped using prophylaxis after treatment with Hemgenix, ending their previous continuous therapy…

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