FDA Grants Rare Pediatric Disease Designation to Editas’ Sickle Cell Therapy

Rare Daily Staff

The U.S. Food and Drug Administration has granted Rare Pediatric Disease designation for EDIT-301, Editas Medicine’s experimental cell therapy for the treatment of sickle cell disease.

Sickle cell disease is an inherited blood disorder caused by a mutation in the beta-globin gene that leads to polymerization of the sickle hemoglobin protein. In sickle cell disease, the red blood cells are misshapen, in a sickle shape instead of the disc shape. The abnormal shape causes the cells to block blood flow causing anemia, pain crises, organ failure, and early death. There are an estimated 100,000 people in the United States currently living with sickle cell disease. Fetal hemoglobin protects against sickle cell disease by inhibiting HbS polymerization.

EDIT-301 is an experimental, autologous cell therapy medicine under investigation for the treatment of sickle cell disease. EDIT-301 is comprised of sickle patient CD34+ cells genetically modified using a highly specific and efficient CRISPR/Cas12a ribonucleoprotein to edit the HBG1/2 promoter region in the beta-globin locus. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin production, which has the potential to provide a durable treatment benefit for people living with sickle cell disease.

Editas plans to file an investigational new drug application for EDIT-301 by the end of 2020.

“The Editas team has a bold vision to unlock the potential of CRISPR to design and develop game-changing medicines,” said Cynthia Collins, CEO of Editas Medicine.  “We know patients are counting on us, and this designation is a significant milestone for the program that highlights the serious, life-threatening manifestations of sickle cell disease.”

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes EDIT-301 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA. There are no approved therapies for the condition.

The vouchers 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, Sarepta sold its priority review voucher to Vifor Pharma $111 million in February 2020.

Photo: Cynthia Collins, CEO of Editas Medicine

Editor’s note: This story was updated to correct the most recent sales of a priority review voucher.