RARE Daily

Editas Scores Rare Pediatric Disease Designation for Beta Thalassemia Therapy

April 26, 2022

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to gene editing therapy developer Editas Medicine for EDIT-301, an experimental therapy for the treatment of the rare blood condition beta thalassemia.

Photo: James Mullen, chairman, president, and CEO of Editas

The FDA previously granted Rare Pediatric Disease designation to EDIT-301 for the treatment of sickle cell disease.

Beta thalassemia is a rare genetic disorder in which 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 creation of blood cells. Based on clinical severity and transfusion requirements, beta thalassemia can be classified into non-transfusion-dependent 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 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 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. 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 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 Mullen, chairman, president, and CEO of Editas. “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. The voucher can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, BioMarin Pharmaceutical sold its voucher for $110 million.

Author: Rare Daily Staff

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