EU Authorizes CSL’s Gene Therapy for Hemophilia B
The authorization is for adult patients with moderately severe and severe Hemophilia B, a rare genetic bleeding disorder caused by a deficiency of the Factor IX protein.
CSL’s application was supported by results from a clinical trial that demonstrated increases in Factor IX activity and a significant reduction in the annualized bleed rate of treated patients.
Hemgenix received FDA’s approval in November 2022.
Etranacogene dezaparvovec
Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of hemophilia B. Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists of a viral vector carrying a gene for clotting Factor IX. The gene is expressed in the liver to produce Factor IX protein, to increase blood levels of Factor IX and thereby limit bleeding episodes. Hemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of blood clotting Factor IX, a protein needed to produce blood clots to stop bleeding.
About CSL Limited
CSL Limited is an Australian multinational specialty biotechnology company that researches, develops, manufactures, and markets products to treat and prevent serious human medical conditions. CSL's product areas include blood plasma derivatives, vaccines, antivenom, and cell culture reagents used in various medical and genetic research and manufacturing applications. CSL was founded in 1916 as the Commonwealth Serum Laboratories, an Australian government body focused on vaccine manufacture.
February 24, 2023https://www.fdanews.com/
