FDA tags Editas' thalassemia cell therapy an orphan drug as it rounds final bend in race to clinic

Editas Medicine has picked up more regulatory privileges for its ex vivo gene-edited medicine, securing FDA orphan-drug designation in beta thalassemia ahead of the start of a clinical trial in the indication.

The candidate, EDIT-301, is in development as a treatment for transfusion-dependent beta thalassemia and sickle cell disease. Editas is advancing the cell therapy in the indications in the belief that mimicking the naturally occurring mutations in patients with hereditary persistence of fetal hemoglobin will restore a healthy level of fetal hemoglobin, thereby freeing beta thalassemia patients from transfusions.

Editas has yet to show that the theory translates to the clinic but, with fewer than 200,000 people in the U.S. suffering from beta thalassemia, it has met the criteria for orphan drug designation. The status positions Editas to receive tax credits, prescription drug user-fee exemptions and marketing exclusivity.

“Receiving orphan drug designation for EDIT-301 for beta thalassemia highlights the urgent need for new treatment options for patients. Preparations to initiate the phase 1/2 clinical trial of EDIT-301 ... are underway, and we look forward to dosing the first patient in the clinical trial this year,” Editas CEO James Mullen said in a statement.

The FDA has authorized a trial that will assess a single intravenous dose of EDIT-301 in adults aged 18 to 50 years. Editas will take CD34+ hematopoietic stem and progenitor cells from a patient, make edits to induce the expression of anti-sickling fetal hemoglobin and readminister the cells into the same patient. Patients will undergo preconditioning during extended stays in hospital.

Editas is one of a small band of companies working on cell therapies for beta thalassemia. Vertex and CRISPR Therapeutics have a gene-edited cell therapy in phase 2/3, and companies including Graphite Bio are giving chase.