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bluebird (BLUE) Filing for Thalassemia Therapy Gets Priority Review

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bluebird bio, Inc. BLUE recently announced that the FDA has accepted the biologics license application (BLA) for betibeglogene autotemcel (beti-cel) for priority review.

The BLA is seeking approval of beti-cel, a potentially transformative gene therapy for adult, adolescent and pediatric patients with β-thalassemia across all genotypes who require regular red blood cell (RBC) transfusions.

The regulatory body has set a target action date of May 20, 2022.

β-thalassemia is a severe genetic disease for those requiring regular RBC transfusions caused by mutations in the β-globin gene, which may cause significantly reduced adult hemoglobin (Hb).

Per the company, a tentative approval will make beti-cel the first one-time treatment that addresses the underlying genetic cause of disease for patients living with β-thalassemia in the United States. The treatment should offer an alternative to regular RBC transfusions and iron chelation therapy that carry the risk of progressive multi-organ damage and increased risk of morbidity and mortality.

The BLA for beti-cel is based on data from phase III studies HGB-207 (Northstar-2) and HGB-212 (Northstar-3), the phase I/II HGB-204 (Northstar) and HGB-205 studies, and the long-term follow-up study LTF-303. As of Mar 9, 2021, the results included a total of 63 pediatric, adolescent and adult patients, including long-term efficacy and safety results in two patients with more than seven years of follow-up.

The FDA earlier granted beti-cel Orphan Drug status and a Breakthrough Therapy designation. It is marketed as Zynteglo in the European Union.

The stock has plunged 75.6% so far this year compared with the industry’s 16.4% decline.

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On Nov 4, 2021, bluebird bio completed the spin-off of its oncology business, 2seventy bio, Inc TSVT.

In collaboration with Bristol Myers BMY, 2seventy bio is commercializing Abecma (ide-cel) and developing bb21217 as treatments for multiple myeloma. 2seventy bio is co-developing and co-promoting Abecma in the United States with Bristol Myers and has exclusively licensed the latter the development and commercialization rights for ide-cel outside of the United States.

We note that Bristol Myers won FDA approval for Abecma as a treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 monoclonal antibody in March 2021. 2seventy bio has exclusively licensed the development and commercialization rights for the bb21217 product candidate to Bristol Myers, with an option for 2seventy bio to elect to co-develop and co-promote bb21217 within the United States.

Following the separation, bluebird bio will focus on transformative gene therapies for severe genetic diseases, including transfusion-dependent β-thalassemia, sickle cell disease (SCD) and cerebral adrenoleukodystrophy, or CALD.

In July 2021, bluebird decided to focus its efforts on the United States market for beti-cel, eli-cel, and LentiGlobin for SCD and is currently winding down operations in Europe.

bluebird bio’s clinical studies of elivaldogene autotemcel (eli-cel, Lenti-D) are currently on clinical hold due to diagnoses of myelodysplastic syndrome likely mediated by Lenti-D lentiviral vector insertion. Nevertheless, the company believes it will be able to file the BLA for eli-cel for the treatment of patients with CALD by the end of 2021.

bluebird currently has a Zacks Rank #3 (Hold). A better-ranked stocks in the biotech sector is Sarepta Therapeutics SRPT which carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Loss per share estimates for Sarepta have narrowed from $6.95 to $4.99 for 2021 and from $4.83 to $3.61 for 2022 in the past 30 days. Sarepta delivered an earnings surprise of 11.06%, on average, in the last four quarters.

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