FDA Grants Rare Pediatric Disease Designation for Experimental Beta Thalassemia Drug

EDIT-301 is an investigational, gene editing medicine developed by Editas Medicine for the treatment of beta thalassemia. As recently reported in the Business Insider, EDIT-301 has just been granted the FDA’s Rare Pediatric Disease designation.

Previously, EDIT-301 had been awarded the Rare Pediatric Disease designation to treat sickle cell disorder. The medication is also being investigated for sickle cell disease in the RUBY clinical trial that is currently recruiting.

This development is very exciting for patients with this blood disorder. The CEO of Editas, James Mullen, stated,

“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.”

About Beta Thalassemia

Beta thalassemia is an inherited blood disorder whereby the production of hemoglobin is reduced. This leads to fewer mature red blood cells, less oxygen, and anemia. More severe cases of beta-thalassemia may lead to enlarged spleen and liver. For severe cases, blood transfusions are usually necessary.

According to the FDA, the definition of rare pediatric disorder is “severe or life-threatening” affecting less than 200,000 individuals in the U.S. The disease primarily affects people beginning from birth through age 18.

Edits, the developer, plans to begin a Phase ½ trial of EDIT-301 for beta thalassemia patients who are transfusion-dependent.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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