Review of Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A — Ozelo 2022

This is a brief audio review. In the article Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A published, 2022 by Ozelo and Colleagues, The outcomes and key points are as noted: Valoctocogene roxaparvovec is an adeno-associated virus 5 based gene-therapy vector with a coagulation factor VIII complementary DNA sequence and an associated by a liver-selective promoter, and it was successful in lowering bleeding rates in hemophiliac patients with Factor 8 deficiency In a Phase 3, Randomized control trial, which was an open label, multi-center study, with 134 patients who were male and 18 years old or older, with severe hemophilia type A, defined as factor 8 levels of 1 international unit per deciliter or lower, and also had no history of anti adenovirus 5 antibodies or a history of factor 8 inhibition with previous use of prophylactic factor 8 concentrate.

Ozelo MC, Mahlangu J, Pasi KJ, Giermasz A, Leavitt AD, Laffan M, Symington E, Quon DV, Wang JD, Peerlinck K, Pipe SW, Madan B, Key NS, Pierce GF, O'Mahony B, Kaczmarek R, Henshaw J, Lawal A, Jayaram K, Huang M, Yang X, Wong WY, Kim B; GENEr8-1 Trial Group. Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A. N Engl J Med. 2022 Mar 17;386(11):1013-1025. doi: 10.1056/NEJMoa2113708. PMID: 35294811.