Frontier bleeding rate reduced by 84%, hemophilia A gene therapy Phase 3 clinical results listed on NEJM

▎WuXi AppTec content team editors Today, BioMarin Pharmaceutical announced that the results of the Phase 3 clinical trial of the gene therapy valoctocogene roxaparvovec for the treatment of severe hemophilia A were published in the prestigious medical journal The New England Journal of Medicine
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The company noted that this is the fifth study the company has published in the New England Journal of Medicine in five years
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Hemophilia A is a rare inherited bleeding disorder caused by a deficiency of factor VIII
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The lack of coagulation factors causes insufficient thrombin generation in patients, resulting in coagulation disorders
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The main clinical manifestations of hemophilia A are repeated bleeding and related complications, of which about 80% are joint hemorrhage
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At present, the main treatment for hemophilia A is regular infusion of coagulation factor VIII, but frequent infusions bring great inconvenience to patients' lives
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Valoctocogene roxaparvovec is a gene therapy that uses an AAV5 viral vector to deliver a transgene expressing Factor VIII
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The advantage is that patients may only need to receive a single treatment, and the liver cells can continue to express factor VIII, eliminating the need for long-term prophylactic coagulation factor injections
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Published study results showed that following a single infusion of valoctocogene roxaparvovec, subjects experienced significantly lower annual bleeding rate (ABR), less frequent factor VIII use, and increased factor VIII activity compared to data from the year prior to enrollment
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After 4 weeks of treatment, patients' annual factor VIII use and ABR requiring treatment were reduced by 99% and 84%, respectively (p<0.
001)
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Overall, 90% (121/134) of trial participants had no bleeding events requiring treatment or had fewer bleeding events compared with receiving factor VIII prophylaxis
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"Breakthrough bleeding that persists with preventive treatment is an unmet medical need for many patients
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I am encouraged that 90% of subjects were free of bleeding requiring treatment in the first year after treatment, or fewer bleeding events than those receiving factor VIII prophylaxis
,
" said the clinical trial's principal investigator, Margareth C.
Ozelo, MD, of Campinas University.
"These results reflect the efficacy of this gene therapy for sustained bleeding control in patients with hemophilia A.
" potential
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Reference: [1] BioMarin Announces Publication in New England Journal of Medicine of One-Year Results from Phase 3 Pivotal Trial with Valoctocogene Roxaparvovec Gene Therapy in Adults with Severe Hemophilia A.
Retrieved March 17, 2022, from https://investors.
biomarin.
com/2022-03-17-BioMarin-Announces-Publication-in-New-England-Journal-of-Medicine-of-One-Year-Results-from-Phase-3-Pivotal-Trial-with-Valoctocogene- Roxaparvovec-Gene-Therapy-in-Adults-with-Severe-Hemophilia-A Disclaimer: The WuXi AppTec content team focuses on introducing global biomedical health research progress
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This article is for information exchange purposes only, and the views expressed in this article do not represent WuXi AppTec's position, nor do they represent WuXi AppTec's support or opposition to the views expressed in this article
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This article is also not a treatment plan recommendation
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.