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New Gene Therapy Approval for Treating Hemophilia B
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The US Food and Drug Administration (FDA) granted approval on November 22, 2022, to CLS Behring for its gene therapy, HEMGENIX® (etranacogene dezaparvovec-drlb); EtranaDez, CSL222), for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes. This is the first approved gene therapy for hemophilia B. For full label information and more, visit FDA.gov.
The commercial product is anticipated to be available soon with an expected wholesale acquisition cost of $3.5 million, making it the highest-priced biopharmaceutical on the market today.
Hemgenix (etranacogene dezaparvovec-drlb) was specifically designed to make near-normal blood-clotting ability possible for patients with hemophilia B by addressing the underlying cause of their condition: a faulty gene that causes a deficiency in clotting Factor IX (FIX).
Hemgenix Insights and Commentary from Emerging Therapy Solutions' (ETS) Chief Medical Officer, Dr. Surya Singh
- Hemgenix is now the highest-priced, single-use biopharmaceutical therapy on the market.
- The Institute for Clinical and Economic Review (ICER)'s health benefit price benchmark for etranacogene dezaparvovec, denoting the maximum cost-effective price, is $2.96M, which is 15.5% lower than the list (WAC) price. These results and the methods used to develop it are detailed in their Evidence Report.
- Hemophilia Treatment Centers (HTCs) are a critical group of providers in the roll-out of hemophilia B gene therapies. We expect HTCs to have a variety of approaches for identifying the best candidates for Hemgenix and discussing the treatment with them.
- ETS is implementing a Request-for-Information (RFI) process to identify Programs of Experience for hemophilia B treatment that will be completed within the next several months (many centers are still determining whether they will administer Hemgenix, which is rate-limiting). In the meantime, ETS can negotiate one-time agreements and has existing contractual relationships with many of the centers that will be providing this care.
- We do not anticipate delays in therapy administration due to manufacturing issues for this gene therapy.
- Follow-up data on factor levels, factor use, bleeds, and hospitalizations will be critical for all stakeholders to have access to - both within the context of value-based agreements and outside of them.
References available upon request.
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Emerging Therapy Solutions® (ETS) provides the crucial insights, expert data and analysis, and comprehensive support payers need to manage the immense costs, potential risks and intricate processes associated with cell and gene therapies, transplants, and specialty treatments for other costly and complex conditions. If you have a referral or need more information on this therapy and condition or others, please contact ETS at 877.445.4822 or at info@emergingtherapies.com. |
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