FDA Approves Hemophilia B Gene Therapy, but Its Cost May Pose Access Problems for Payers

The FDA recently approved the fifth gene therapy to gain FDA approval, with three of those decisions coming in the second half of this year. While payer respondents to a Zitter Insights survey have expressed interest in the drug, its price may prove to be an obstacle to coverage.

On Nov. 22, the FDA approved uniQure N.V.’s Hemgenix (etranacogene dezaparvovec-drlb) for the treatment of people at least 18 years old 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. CSL Behring LLC, a CSL business, will market the gene therapy. The agency gave the first-in-class adeno-associated virus A5-based gene therapy — which previously was known as EtranaDez — priority review and orphan and breakthrough therapy designations.

While the drug is the first gene therapy for hemophilia B to gain FDA approval, it could be joined by a competitor next year: Pfizer Inc. also is developing a novel gene therapy to treat moderate to severe hemophilia B. Currently in Phase III trials, fidanacogene elaparvovec contains an adeno-associated virus capsid and a high-activity human coagulation factor IX gene.

For the Managed Care Biologics and Injectables Index: Q3 2022, from Aug. 26, 2022, to Sept. 30, 2022, Zitter Insights polled 35 commercial payers covering 121.8 million lives and 27 Medicare payers representing 40.2 million lives about their management of gene therapies. Payers covering 52% of commercial lives and 69% of Medicare ones said they were somewhat aware of Hemgenix. In addition, payers with almost 80% of commercial lives and more than half of Medicare beneficiaries said they had moderate interest in the product (see chart).

Respondents covering approximately 40% of lives stated that they expect the drug to have a high impact on products currently available to treat hemophilia B, with some agents likely becoming not covered and/or having greater use of utilization management criteria.

When asked about the anticipated annualized net price of Hemgenix, respondents said that somewhere between around $670,000 and $690,000 would be appropriately priced. The agent would be considered too expensive — meaning that they would recommend that the product not be covered or be covered in a highly restricted fashion — at a price between $2.0 and $2.1 million.

CSL has revealed that the one-time treatment of Hemgenix via intravenous infusion will cost $3.5 million, making it the most expensive FDA-approved drug available.

For more information on the Zitter Insights data, contact Jill Brown Kettler at jbrown@aishealth.com.

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