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bluebird's (BLUE) Gene Therapy for SCD Gets Priority Review
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bluebird bio, Inc. (BLUE - Free Report) announced that the FDA has accepted the biologics license application (BLA) for its gene therapy lovotibeglogene autotemcel (lovo-cel) for priority review.
The regulatory body has set a target action date of Dec 20, 2023.
Lovo-cel is a potentially transformative, one-time gene therapy for individuals with sickle cell disease (SCD) ages 12 and older who have a history of vaso-occlusive events (VOEs).
The FDA generally grants Priority Review to therapies that are likely to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. The designation comes with various benefits, like a review timeline of six months from the time of filing compared with a standard review timeline of 10 months.
The BLA for lovo-cel is based on efficacy results from 36 patients in the HGB-206 study Group C cohort with a median 32 months of follow-up and two patients in the HGB-210 study with 18 months of follow-up each. The BLA submission also includes safety data from 50 patients treated across the entire lovo-cel program, including six patients with six or more years of follow-up, which is the longest follow-up of any gene therapy program for SCD.
A potential approval will make lovo-cel – bluebird’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.
The FDA previously granted orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation and rare pediatric disease designation to lovo cell.
Shares of BLUE have plunged 50.7% so far this year compared with the industry’s 7.7% decline.
Image Source: Zacks Investment Research
The FDA approved Zynteglo (betibeglogene autotemcel) for the treatment of beta-thalassemia in adult and pediatric patients requiring regular red blood-cell transfusions and Skysona (elivaldogene autotemcel) for the treatment of early, active cerebral adrenoleukodystrophy on Aug 17, 2022 and Sept 16, 2022, respectively.
bluebird has made significant progress in the launch of Zynteglo with six patient starts (cell collections) for patients with beta-thalassemia. The first commercial infusion has been completed and revenues from Zynteglo are expected to be recognized in the second quarter.
The first commercial infusion for Syksona was completed in March 2023. Cell collection has been completed for three patients to be treated with Skysona. bluebird continues to anticipate 5-10 patient starts this year.
Zacks Rank and Stocks to Consider
bluebird currently has a Zacks Rank #3 (Hold). Some better-ranked stocks in the healthcare sector are Ligand Pharmaceuticals (LGND - Free Report) and Novartis (NVS - Free Report) . LGND currently sports a Zacks Rank #1 (Strong Buy) and Novartis carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, earnings estimates for LGND have increased by 86 cents per share to $5.25. LGND topped earnings estimates in two of the last four quarters and missed in the remaining two, the average surprise being 21.50%.
Over the past 60 days, NVS’ earnings estimates have increased to $6.74 from $6.57 for 2023. Novartis surpassed estimates in all the trailing four quarters, the average surprise being 5.15%.
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bluebird's (BLUE) Gene Therapy for SCD Gets Priority Review
bluebird bio, Inc. (BLUE - Free Report) announced that the FDA has accepted the biologics license application (BLA) for its gene therapy lovotibeglogene autotemcel (lovo-cel) for priority review.
The regulatory body has set a target action date of Dec 20, 2023.
Lovo-cel is a potentially transformative, one-time gene therapy for individuals with sickle cell disease (SCD) ages 12 and older who have a history of vaso-occlusive events (VOEs).
The FDA generally grants Priority Review to therapies that are likely to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. The designation comes with various benefits, like a review timeline of six months from the time of filing compared with a standard review timeline of 10 months.
The BLA for lovo-cel is based on efficacy results from 36 patients in the HGB-206 study Group C cohort with a median 32 months of follow-up and two patients in the HGB-210 study with 18 months of follow-up each. The BLA submission also includes safety data from 50 patients treated across the entire lovo-cel program, including six patients with six or more years of follow-up, which is the longest follow-up of any gene therapy program for SCD.
A potential approval will make lovo-cel – bluebird’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.
The FDA previously granted orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation and rare pediatric disease designation to lovo cell.
Shares of BLUE have plunged 50.7% so far this year compared with the industry’s 7.7% decline.
Image Source: Zacks Investment Research
The FDA approved Zynteglo (betibeglogene autotemcel) for the treatment of beta-thalassemia in adult and pediatric patients requiring regular red blood-cell transfusions and Skysona (elivaldogene autotemcel) for the treatment of early, active cerebral adrenoleukodystrophy on Aug 17, 2022 and Sept 16, 2022, respectively.
bluebird has made significant progress in the launch of Zynteglo with six patient starts (cell collections) for patients with beta-thalassemia. The first commercial infusion has been completed and revenues from Zynteglo are expected to be recognized in the second quarter.
The first commercial infusion for Syksona was completed in March 2023. Cell collection has been completed for three patients to be treated with Skysona. bluebird continues to anticipate 5-10 patient starts this year.
Zacks Rank and Stocks to Consider
bluebird currently has a Zacks Rank #3 (Hold). Some better-ranked stocks in the healthcare sector are Ligand Pharmaceuticals (LGND - Free Report) and Novartis (NVS - Free Report) . LGND currently sports a Zacks Rank #1 (Strong Buy) and Novartis carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, earnings estimates for LGND have increased by 86 cents per share to $5.25. LGND topped earnings estimates in two of the last four quarters and missed in the remaining two, the average surprise being 21.50%.
Over the past 60 days, NVS’ earnings estimates have increased to $6.74 from $6.57 for 2023. Novartis surpassed estimates in all the trailing four quarters, the average surprise being 5.15%.