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FDA Approves $3.5M Drug Used for Hemophilia B

There is a new “most expensive drug” on the market in the United States.

On November 22, 2022, Hemgenix was granted FDA approval for the treatment of adults with Hemophilia B. The gene therapy manufactured by CSL Behring LLC has a projected cost of $3.5 million per treatment, topping the previous most expensive drug – Zolgensma, which is used to treat spinal muscular atrophy – by 67%.

Hemophilia B only represents 15% of all patients with hemophilia. The prevalence of hemophilia B is 1 in 40,000 Americans, with most individuals experiencing symptoms being men. Hemgenix is a one-time treatment given by IV infusion. Safety and effectiveness were evaluated in two studies conducted with adult men experiencing severe or moderately severe symptoms. If clinical approval correlates with specific trial exclusion criteria, roughly 60% of those suffering from hemophilia B would not qualify for this extremely specialized treatment.

The ICER, or Institute for Clinical and Economic Review, has evaluated and considered Hemgenix’s list price as cost-effective. The life-long cost estimate for Hemophilia B can reach up to $23 million for prophylactic treatment in adults. The ICER noted that “prophylactic treatment… itself is widely considered to be far too expensive to be cost-effective.” The ICER’s fair pricing benchmark for Hemgenix was found to be between $2.93 million to $2.96 million.

Regarding the pharmacy benefit, Pharmacy Benefit Managers (PBMs) have not yet received the drug file in their database for P&T (Pharmacy & Therapeutics Committee) review. Details on formulary coverage, clinical criteria, and pricing cannot be made available until these reviews are complete. Plan sponsors are encouraged to review their current coverage of gene therapies and explore strategies for cost containment.

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