UntilAugust 2019, 22 gene medicines had been approved by the drug regulatory agencies from various countries, butthere were few relevant reviews of combing these drugs systematically. Consequently, this review summarizesthe gene therapy drugs approved worldwide from 1998 to 2019 in details, including ...
With the development of various gene therapy technologies, the safety and effectiveness of the carrier used for gene therapy products have been gradually improved, and a number of gene therapy products have been approved for marketing. Table 1 Gene therapy drugs approved for marketing in the US ...
Most experts in the medical field will tell you that gene therapy has finally come of age, but the numbers tell a different story. Despite 30 years of research and a bigger pipeline than ever, only a small number of gene therapy trials have completed lat
doi:10.1007/s40267-023-00986-2Yu D.Springer International PublishingDrugs & therapy perspectives: for rational drug selection and use
Valoctocogene roxaparvovec, a one-time, single-dose IV infusion, is the first gene therapy approved in the United States for severe hemophilia A and will cost around $2.9 million. BioMarin has said theprice tag reflects"the possibility of freedom from years" of infusions, which come to ab...
subretinal injections), and peripheral intramuscular injection [33,34,35]. The research objective of in vivo gene therapy is to enhance the precision, efficacy, and long-term effectiveness of gene therapy. Presently, the majority of approved gene therapy drugs predominantly employ in vivo administrati...
FDA approved Elevidys, the first gene therapy for the treatment of pediatric patients 4 through 5 years of age with Duchenne muscular dystrophy (DMD)
The primary report of the EMBARK phase 3 trial, testing the AAV-based gene therapy delandistrogene moxeparvovec in Duchenne muscular dystrophy, did not meet its primary endpoint of improvement in NSAA mobility scores compared to placebo. Secondary endpoints show that the therapy was safe and asso...
Therapy, CBER director Peter Marks said speeding up the withdrawal of approved gene therapies could be easier and faster than for small-molecule therapies. At the same time, he stressed that "accelerated approval of gene therapy is not a get-out-of-jail-free ticket." The FDA will not ...
The ACE Model would apply to Part B drugs approved through the AAP Program. In its recent update, CMS notes that “more than 90% of accelerated approvals for Part B drugs over the past five years were for oncology indications,” although this model could also...