Editas Medicine Receives FDA Rare Pediatric Disease Designation for EDIT-301 for the Treatment of Beta Thalassemia

About this update from Editas Medicine, Inc.

[{"type":"text","content":"CAMBRIDGE, Mass., April 26, 2022 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease designation to EDIT-301, an investigational, gene-edited medicine for the treatment of beta thalassemia. The FDA previously granted Rare Pediatric Disease designation to EDIT-301 for the treatment of sickle cell disease. “Beta thalassemia is a devastating disease that leads to severe anemia, organ failure, and premature death. Receiving Rare Pediatric Disease designation for EDIT-301 for beta thalassemia highlights the dire need for new treatment options,” said James C. Mullen, Chairman, President, and Chief Executive Officer, Editas Medicine. “EDIT-301 is a potentially transformative medicine for patients living with beta thalassemia, and we look forward to dosing the first patient in our clinical trial this year.” The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals aged from birth to 18 years. Pediatric diseases recognized as “rare” affect fewer than 200,000 people in the United States. Under the FDA’s Rare Pediatric Disease Designation and Voucher Programs, if Editas receives marketing approval for EDIT-301 for beta thalassemia, the Company may be eligible to receive a Priority Review Voucher (PRV) from the FDA that can be redeemed to receive priority review of a subsequent marketing application for a different product, or the PRV may be sold or transferred. EDIT-301 is being investigated in a clinical study in patients with severe SCD (RUBY trial, NCT04853576). Editas expects to initiate a Phase 1/2 study of EDIT-301 in patients with transfusion-dependent beta thalassemia in 2022. About Beta Thalassemia Beta thalassemia is a common autosomal recessive disorder with an estimated annual incidence rate of 1 in 100,000 worldwide for symptomatic individuals. Beta thalassemia mutations reduce or abrogate beta globin expression. Insufficient beta globin production leads to ineffective red blood cell production, chronic hemolytic anemia due to the destruction of red blood cells, and compensatory extramedullary hematopoiesis (creation of blood cells). Based on clinical severity and transfusion requirements, beta thalassemia...

More updates from Editas Medicine, Inc.