HEMOPHILIA GENE THERAPY MARKET ANALYSIS

Market News

Global Hemophilia Gene Therapy Market: Key Developments

In July 2022, Sanofi, pharmaceutical and healthcare company, and Swedish Orphan Biovitrum, biopharmaceutical company collaborate on the development and commercialization of efanesoctocog alfa. The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to efanesoctocog alfa (BIVV001) for the treatment of people with hemophilia A, a rare and life-threatening bleeding disorder, based on data from the pivotal XTEND-1 Phase 3 study. Efanesoctocog alfa is the first factor VIII therapy to be awarded Breakthrough Therapy designation by the FDA. Efanesoctocog alfa is a novel and investigational factor VIII therapy designed to provide normal to near-normal factor activity levels for the majority of the week in a once-weekly prophylactic treatment regimen. Breakthrough Therapy designation is designed to expedite the development and review of drugs in the U.S. that target serious or life-threatening conditions. Drugs qualifying for this designation must show preliminary clinical evidence that the drug may demonstrate a substantial improvement on clinically significant endpoints over available therapies.

In December 2018, Spark Therapeutics announced updated preliminary data for the ongoing phase 1/2 clinical trial of investigational SPK-8011 in hemophilia A.

In January 2021, the League of Clinical Research, Russia carried out Interventional study for Efmoroctocog Alfa Injection [Eloctate] to treat haemophilia A. The study is currently in Phase 4 and the study is estimated to be completed on November 15, 2022.