New Drug Capsule: etranacogene dezaparvovec

Abstract

Generic name: etranacogene dezaparvovec1FDA news release. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treat-adults-hemophilia-b. Current as of 11//22/2022. Retrieved December 7, 2022.Google Scholar Brand name: Hemgenix Manufacturer: CSL Behring LLC Class: gene therapies; antihemorrhagics; blood coagulation factors Date of FDA Approval: November 22, 2022 Cost: US$3.5 million2Naddaf M. Researchers welcome $3.5 million haemophilia gene therapy – but questions remain. Nature. https://www.nature.com/articles/d41586-022-04327-7. Posted December 6, 2022. Retrieved December 7, 2022.Google Scholar For the treatment of adults with hemophilia B (congenital factor IX deficiency) who currently use factor IX prophylaxis therapy or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes. Etranacogene dezaparvovec (Hemgenix) consists of an adeno-associated 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. The most common adverse reactions include liver enzyme elevations, headache, mild infusion-related reactions, and influenza-like symptoms. Patients should be monitored for adverse infusion reactions and liver enzyme elevations (transaminitis) in their blood. One-time gene therapy product given as a single dose by intravenous infusion. The safety and effectiveness were evaluated in 2 studies of 57 men with severe or moderately severe hemophilia B receiving prophylactic factor IX replacement therapy. Effectiveness was established based on decreases in the men's annualized bleeding rate (ABR). In the Phase III HOPE-B (Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients) study, which had 54 participants, a single dose of etranacogene dezaparvovec increased factor IX activity levels (39% at 6 months and 36.7% at 24 months after infusion), reduced the mean ABR compared with baseline by 54%, and reduced the need for routine factor IX replacement in 94% of patients.3CSL Behring. Final analysis of pivotal HOPE-B study demonstrates durable and sustained therapeutic effect of etranacogene dezaparvovec gene therapy in hemophilia b – data presented at EAHAD 2022 [media release]. February 4, 2022. https://www.cslbehring.com/.Google Scholar,4CSL Behring. CSL's novel gene therapy HEMGENIX® (etranacogene dezaparvovec-drlb) demonstrates durable protection and sustained factor IX activity levels for people living with hemophilia B at 24-months post-treatment [media release]. December 10, 2022. https://www.cslbehring.com/.Google Scholar Hemophilia B is a genetic bleeding disorder that results from missing or insufficient levels of blood clotting factor IX, a protein needed to produce blood clots to stop bleeding. Symptoms can include prolonged or heavy bleeding after an injury, surgery, or dental procedure; in severe cases, bleeding episodes can occur spontaneously without a clear cause. Prolonged bleeding episodes can lead to serious complications, such as bleeding into joints, muscles, or internal organs, including the brain. Most individuals who have hemophilia B and experience symptoms are men. The prevalence of hemophilia B in the population is approximately 1 in 40,000; hemophilia B represents approximately 15% of patients with hemophilia. Many female carriers of the disease have no symptoms. However, an estimated 10% to 25% of women carriers have mild symptoms; in rare cases, women may have moderate or severe symptoms. Treatment typically involves replacing the missing or deficient clotting factor to improve the body's ability to stop bleeding and promote healing. Patients with severe hemophilia B typically require a routine treatment regimen of infusions of factor IX replacement products to maintain sufficient levels of clotting factor to prevent bleeding episodes. This application received priority review, orphan, and breakthrough therapy designations.