DTMT Network

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Editas Medicine’s EDIT-301, an experimental gene-editing medicine, for the treatment of beta-thalassemia.

In a statement released recently, the company informed that the drug had earlier received Rare Paediatric Disease designation from the drug regulator for the treatment of beta-thalassemia and sickle cell disease.

Beta thalassemia is a common genetic disorder estimated to occur among one in 100,000 people across the world every year and results in reduced beta globin production, leading to ineffective red blood cell production, chronic haemolytic anaemia due to the destruction of red blood cells, and compensatory extramedullary creation of blood cells.

“Beta-thalassemia is a devastating disease that leads to severe anaemia, organ failure, and premature death,” James C. Mullen, Chairman, President and Chief Executive Officer of Editas Medicine said.

“Receiving Orphan Drug Designation for EDIT-301 for beta-thalassemia highlights the urgent need for new treatment options for patients,” he added.

The company is making preparations to start the first phase of the two-phase clinical trial of the drug during the current year, EDIT-301, Mullen said.

It may be noted that medicines that are granted Orphan Drug Designation entitle the developers to certain incentives, including tax credits for qualified clinical trials, prescription drug user-fee exemptions, and potential seven-year marketing exclusivity once they receive approval from FDA.

Editas said that the red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in foetal haemoglobin production, which has the potential to provide a one-time, durable treatment benefit for people suffering from severe sickle cell disease and transfusion-dependent beta-thalassemia.