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

Bluebird Bio Inc, a prominent biotechnology company traded on the NASDAQ under the ticker BLUE, has recently submitted a comprehensive Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking approval for its groundbreaking gene therapy known as lovotibeglogene autotemcel, or lovo-cel, to be used in patients aged 12 and older who suffer from sickle cell disease (SCD) and have a history of vaso-occlusive events (VOEs). By accompanying the BLA with a request for Priority Review, Bluebird Bio aims to expedite the FDA’s review process, with the potential to shorten it to just six months as opposed to the standard timeline of 10 months.


If the FDA grants approval, it will mark a significant milestone for Bluebird Bio as lovo-cel would become their third ex-vivo gene therapy to be approved for the treatment of a rare genetic disease. Notably, it would also be their second FDA approval for addressing an inherited hemoglobin disorder. The decision to delay the submission of the sickle cell disease drug application to the FDA earlier this year had raised questions about the company’s progress. However, this recent BLA submission signals significant progress in their efforts to bring this promising therapy to patients in need.


In the realm of sickle cell disease gene therapies, it is important to acknowledge the efforts of CRISPR Therapeutics AG (CRSP) and Vertex Pharmaceuticals Incorporated (VRTX). These two companies have made significant advancements in the development of ex-vivo cell therapy, specifically exa-cel, which is intended for the treatment of sickle cell disease and transfusion-dependent beta-thalassemia. While it is widely expected that CRISPR/Vertex will be the first to reach the market with a gene therapy for sickle cell disease, it is worth noting that the time advantage they hold over Bluebird Bio may not be as substantial as initially anticipated due to the closer proximity of the BLA filings. As a result, the competition in the gene therapy market for sickle cell disease treatment is likely to become more intense and dynamic.


In a promising turn of events, there is now a possibility of a joint advisory committee being formed to specifically address gene therapies for sickle cell disease. This committee would bring together esteemed experts in the field to provide guidance and recommendations concerning the development, regulation, and implementation of gene therapy treatments for this debilitating disease. The formation of such a committee could potentially help streamline the regulatory process, foster collaboration among industry stakeholders, and ensure the safe and effective use of these transformative therapies.


Furthermore, the Institute for Clinical and Economic Review (ICER), an independent organization that evaluates the value and affordability of healthcare interventions, has scheduled a meeting on July 27 to discuss the cost-effectiveness of both lovo-cel and exa-cel for the treatment of sickle cell disease. The ICER meeting will focus on assessing the clinical benefits, long-term outcomes, and pricing considerations associated with these gene therapies. This evaluation will play a crucial role in determining their economic impact and overall value proposition.


In summary, Bluebird Bio has submitted a BLA to the FDA for the approval of their gene therapy, lovo-cel, for SCD patients aged 12 and above with a history of VOEs. They have requested Priority Review, aiming for a faster approval process. Although CRISPR/Vertex is expected to be the first to market, the proximity of the BLA filings suggests that Bluebird Bio may not be far behind. The establishment of a joint advisory committee and the upcoming ICER meeting further emphasize the significance of gene therapies in treating SCD.


Reference: Bluebird scd