With FDA Submission For Sickle Cell Gene Therapy, Bluebird Bio Can Potentially Have 3 Approved Products By 2023

Bluebird Bio Inc BLUE has submitted its Biologics License Application (BLA) to the FDA seeking approval for lovotibeglogene autotemcel (lovo-cel) gene therapy in patients with sickle cell disease (SCD) ages 12 and older who have a history of vaso-occlusive events (VOEs).
The BLA includes a request for Priority Review, which, if granted, would shorten the FDA's review of the application to six months versus a standard review timeline of 10 months.
If approved, lovo-cel will be bluebird bio's third ex-vivo gene therapy approved by the FDA for a rare genetic disease and its second FDA approval for an inherited hemoglobin disorder.
In March, Bluebird Bio signaled a delay in its sickle cell disease drug application to the FDA.
William Bliar notes that earlier this month, CRISPR Therapeutics AG CRSP and Vertex Pharmaceuticals Incorporated VRTX completed rolling BLA submission for their ex-vivo cell therapy, exa-cel, for the treatment of SCD and transfusion-dependent beta-thalassemia.
The analyst notes that although CRISPR/Vertex will likely still be first to market with gene therapy for SCD, their lead time on Bluebird will not be as long as initially thought, given the closer proximity of the BLA filings.
Furthermore, a joint advisory committee on gene therapies for SCD is possible now.
Separately, the Institute for Clinical and Economic Review (ICER) will hold a meeting on July 27 to discuss the price effectiveness of lovo-cel and exa-cel for SCD.
Related: $2M Price Tag for Sickle Cell Gene Therapies Could Be Cost-Effective Under Certain Scenarios, Finds ICER Analysis.
Price Action: BLUE shares are down 0.29% at $3.42 on the last check Monday.

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