bluebird bio's Rare Blood Disorder Drug Tagged a Breakthrough by the FDA

Published: Feb 03, 2015

bluebird bio's Rare Blood Disorder Drug Tagged a Breakthrough by the FDA
February 2, 2015
By Krystle Vermes, Breaking News Staff

Gene therapy developer bluebird bio , announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to LentiGlobin, its drug product for the treatment of transfusion-dependent patients with beta-thalassemia major.

The LentiGlobin BB305 Drug Product is also aimed at treating severe sickle cell disease by inserting a human beta-globin gene into the patient’s own hematopoietic stem cells ex vivo. Afterward, it returns those modified cells to the patient through autologous stem cell transplantation.

“The FDA's Breakthrough designation of LentiGlobin highlights that new therapies are needed for the treatment of patients with beta-thalassemia major, especially treatments with the potential to meaningfully reduce or liberate patients from transfusion dependence,” said David Davidson, chief medical officer of bluebird bio.

The Breakthrough Therapy designation will expedite the development and review of the drug candidate. This designation is only given to products that have the potential to treat serious or life-threatening illnesses. The drugs must also have preliminary clinical evidence that shows that it may provide substantial improvement over existing therapies.

"Our early clinical data investigating the use of LentiGlobin in patients with multiple genotypes of beta-thalassemia major, including beta-0/beta-0, the most severe genotype, are very encouraging, and we remain on track to complete enrollment in the Northstar and HGB-205 studies in 2015,” Davidson continued. “In light of the Breakthrough designation, we look forward to working even more closely with the FDA to expedite the development of LentiGlobin for the treatment of beta-thalassemia major."

bluebird Releases Promising Data
In December 2014, bluebird bio released data from eight subjects that had been treated with LentiGlobin BB305. In the first four patients, the drug was able to provide sufficient hemoglobin production to reduce or eliminate the need for transfusion. This data was presented alongside results from the Phase I/II Northstar study at the 56th Annual Meeting of the American Society of Hematology.

“Beta-thalassemia major is a devastating disease that affects 40,000 newborn children globally every year, and the existing treatment options for these patients have significant side effects and limitations,” said Alexis Thompson, lead investigator of the Northstar Study. “Data from the Northstar Study presented today further demonstrate the potential for a one-time gene therapy treatment to transform the lives of patients with beta-thalassemia major, including those with the most severe genotype of beta-thalassemia major, beta-0/beta-0, also known as Cooley’s Anemia.”

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