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

Editas Medicine, Inc. recently announced  US 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 US.

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.

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 can be classified into non-transfusion-dependent (NTDT) and transfusion-dependent beta thalassemia (TDT). TDT is the most severe form of beta thalassemia, and patients require lifelong regular red blood cell transfusions to prevent organ failure and death. Chronic red blood cell transfusions are complicated by iron overload leading to organ dysfunction and failure. Left untreated, the mortality rate among TDT patients is high, with a survival rate of only 15 percent at age five due to severe anemia.

EDIT-301 is an experimental cell therapy medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). EDIT-301 consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, where naturally occurring fetal hemoglobin (HbF) inducing mutations reside, by a highly specific and efficient proprietary engineered AsCas12a nuclease. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin production, which has the potential to provide a one-time, durable treatment benefit for people living with severe sickle cell disease and transfusion-dependent beta thalassemia.

As a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For more information, visit www.editasmedicine.com.