Gene/Cell Therapy Quarterly Update: December 2022

This quarterly pipeline wrap-up provides a review of newly approved gene and cell therapies, new indications and news of note on gene and cell therapies drugs in the approval process. See other articles for updates on specialtydrugs, biosimilar drugs and  traditional drugs. 

New Drug Information

• Hemgenix^® (etranacogene dezaparvovec-drlb): The U.S. Food and Drug Administration (FDA) has approved CSL Behring’s Hemgenix for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes. Hemgenix is a one-time gene therapy product given as a single dose by IV infusion. In one study, patients who received Hemgenix had increases in factor IX activity levels, a decreased need for routine factor IX replacement prophylaxis, and a 54% reduction in annualized bleeding rate (ABR) compared to baseline.¹ In a recent cost-effectiveness analysis of the drug, weighing health benefits against offset costs, the Institute for Clinical and Economic Review (ICER), an independent nonprofit research organization suggested that a fair price for the drug to be between $2.93 million and $2.96 million.² Hemgenix has launched with a wholesale acquisition cost (WAC) of $3.5 million per one-time dose.

New Indications

• None

References

1. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treat-adults-hemophilia-b
2. https://icer.org/news-insights/press-releases/icer-publishes-evidence-report-on-gene-therapies-for-hemophilia-a-and-b/