RARE Daily

FDA’s Requirements Deal Set Back to Bluebird’s Plans for SCD Gene Therapy

November 5, 2020

Rare Daily Staff

In its third quarter 2020 financial results report, Bluebird Bio said that after discussion with the U.S. Food and Drug Administration, it will delay submission of its application to market LentiGlobin for sickle cell disease until late 2022 due to an additional requirement of comparability data to support the submission.

The FDA and Bluebird agreed on the company’s path to transition to commercial manufacturing using an analytical comparability strategy, including suspension-based lentiviral vector.  Specifically, Bluebird said the FDA requested “the use of drug product manufactured from sickle cell disease (SCD) patient cells in addition to healthy donors as well as commercial lentiviral vector to demonstrate drug product comparability,” the company noted.

Bluebird said the requirement will “meaningfully de-risk the bb1111 program and bring clarity on the path to approval.” But, due to the impact of COVID-19 on contract manufacturing organizations, which are experiencing four to six month delays, it will need at least a year’s time to get the required drug product.

LentiGlobin for SCD isn’t Bluebird’s first time dealing with regulatory requirements around commercial manufacturing and quality control issues. Bluebird delayed for six months the commercial launch of its gene therapy Zynteglo, a one-time treatment for certain patients 12 years and older with transfusion-dependent beta-thalassemia, to deal with commercial specifications. The company said it is on track to complete the rolling application submission in the United States in mid-2021.

Bluebird bio shares fell 13.6 percent on the news in pre-market trading.   

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