Gene Therapy
FDA news: Breast cancer, sickle cell disease therapies receive priority review
The FDA announced several regulatory actions the past few weeks.
FDA approves Roctavian for treatment of adults with severe hemophilia A
FDA approved valoctocogene roxaparvovec-rvox for the treatment of certain adults with severe hemophilia A, according to the agent’s manufacturer.
Gene therapy for inherited cardiomyopathy gains FDA fast track, orphan drug designation
Rocket Pharmaceuticals announced that the FDA has granted fast track and orphan drug designations to its adeno-associated virus-based gene therapy for the treatment of plakophilin-2-related arrhythmogenic cardiomyopathy.
FDA approves Vyjuvek for dystrophic epidermolysis bullosa
The FDA has approved Vyjuvek (beremagene geperpavec-svdt), an investigational non-invasive, topical, redosable gene therapy for the treatment of dystrophic epidermolysis bullosa, Krystal Biotech announced in a press release.
Gene therapy a potential ‘functional cure’ for sickle cell disease, beta thalassemia
Treatment with exagamglogene autotemcel conferred clinically meaningful increases in fetal hemoglobin and total hemoglobin levels among patients with beta-thalassemia and sickle cell disease, results of two pivotal studies showed.
Increases in myelin, white matter reported at 6 months with Canavan disease gene therapy
Myrtelle Inc. has reported promising 6-month trial data for a recombinant adeno-associated virus vector-based investigational gene therapy for Canavan disease.
FDA grants fast track designation for novel Duchenne muscular dystrophy gene therapy
Regenxbio Inc. announced that the FDA has granted fast track designation for RGX-202, a one-time gene therapy for treatment of Duchenne muscular dystrophy.
Lumevoq gene therapy shows sustained efficacy, safety at 3 years in phase 3 LHON trial
A phase 3 clinical trial demonstrated positive 3-year results for bilateral intravitreal injection of Lumevoq gene therapy, with statistically significant visual acuity improvements from baseline in both treated eyes.
Valoctocogene roxaparvovec shows durable factor VIII activity in hemophilia A
Valoctocogene roxaparvovec conferred sustained bleeding control at 2 years among a cohort of men with severe hemophilia A, according to phase 3 study results published in The New England Journal of Medicine.
Gene therapy ‘clearly superior’ for patients with hemophilia B
Etranacogene dezaparvovec conferred superior bleed control for adults with moderate or severe hemophilia B compared with standard factor IX prophylaxis, according to results of the phase 3 HOPE-B study.