Global biotherapeutics leader CSL Behring announced that the Committee for Medicinal Products for Human Use (CHMP), the chief scientific body of the European Medicines Agency (EMA) accepted its request for an accelerated assessment of the etranacogene dezaparvovec Marketing Authorisation Application (MAA). Etranacogene dezaparvovec (also known as EtranaDez), currently being studied in the Phase 3 HOPE-B clinical trial, is an investigational gene therapy for people living with hemophilia B, a life-threatening bleeding disorder.
The CHMP grants accelerated assessment when a medicinal product is expected to be of major public health interest, particularly pertaining to therapeutic innovation. Accelerated assessment potentially reduces the review timeline from 210 days to 150 days once the MAA is filed and validated.
“CSL Behring has been dedicated to improving the lives of patients with Hemophilia B for decades and the acceptance of an accelerated regulatory review underscores the high unmet need for a long-term, innovative treatment option for these patients,” said Emmanuelle Lecomte Brisset, Head of Global Regulatory Affairs at CSL Behring. “We look forward to submitting our Marketing Authorization Application in the first half of 2022 so that we can make this novel therapy option available to patients as soon as possible.”
Brahm Goldstein, MD, MCR, Vice President, Research and Development, Hematology added, “Accelerated regulatory review supports our commitment to advanced research and providing pioneering treatment options. Gene therapy, and its associated near-normal blood clotting potential, could be life changing for people living with hemophilia B who are vulnerable to spontaneous bleeding in their muscles, internal organs and joints. This milestone underscores our excitement about the promise of gene therapy for people living with hemophilia B.”
Along with global partner, uniQure, the company recently announced positive topline results from the HOPE-B pivotal trial of etranacogene dezaparvovec. The study achieved its primary endpoint of non-inferiority in annualized bleeding rate after stable Factor IX (FIX) expression, assessed at 18 months following a single dose.