Valoctocogene Roxaparvovec and Etranacogene Dezaparavovec: Novel Gene Therapies for Hemophilia A and B.
| Autor: | Dougherty JA; Lloyd L. Gregory School of Pharmacy, Palm Beach Atlantic University, West Palm Beach, FL, USA., Dougherty KM; Department of Biology, Palm Beach Atlantic University, West Palm Beach, FL, USA. |
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| Jazyk: | angličtina |
| Zdroj: | The Annals of pharmacotherapy [Ann Pharmacother] 2024 Aug; Vol. 58 (8), pp. 834-848. Date of Electronic Publication: 2023 Nov 17. |
| DOI: | 10.1177/10600280231202247 |
| Abstrakt: | Objective: To review efficacy and safety data of valoctocogene roxaparvovec (Roctavian) and etranacogene dezaparavovec (Hemgenix), novel gene therapies for the treatment of the life-threatening bleeding disorders hemophilia A and B, respectively. Data Sources: A PubMed/Google Scholar search from inception through August 11, 2023 was conducted using the following keywords: gene therapy , hemophilia A , hemophilia B , etranacogene dezaparavovec , valoctocogene roxaparvovec , and bleeding. Study Selection and Data Extraction: Data, including phase 1 to 3 clinical trials (non-comparator), were obtained from primary literature and package inserts. These reports evaluated clinical pharmacology, efficacy, safety, adverse events, warnings, and precautions. Data Synthesis: Valoctocogene phase 3 study in males (n = 134): 87% had factor VIII (FVIII) levels that at least met criteria for mild hemophilia. Etranacogene phase 3 study in males (n = 54): within 3 weeks of infusion, mean factor IX (FIX) levels had reached 26.8 IU/dL. Both therapies provided clinically and statistically significant decreases in bleeding events and prophylactic factor infusions. Most common adverse event was elevations in liver function tests that were treated with glucocorticoids. Relevance to Patient Care and Clinical Practice in Comparison With Existing Drugs: The endogenous production of clotting factors mimics physiological production while decreasing morbidity and mortality related to bleeding events similar to the effects of existing replacement strategies. Gene therapy was also shown to increase patient quality of life. Conclusion: Valoctocogene and etranacogene provide another treatment for selected patients with hemophilia. Treatment for the patient with hemophilia (gene therapy vs replacement strategy) must be personalized as new clinical data are published being cognizant of drug affordability. Competing Interests: Declaration of Conflicting InterestsThe authors declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article. |
| Databáze: | MEDLINE |
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