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bluebird's (BLUE) Gene Therapy Gets FDA Nod for Beta-Thalassemia
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bluebird bio (BLUE - Free Report) recently announced that the FDA has approved Zynteglo (betibeglogene autotemcel), as a one-time gene therapy for treating beta-thalassemia, in adult and pediatric patients who require regular red blood cell (RBC) transfusions
Beta-thalassemia is a severe genetic blood disease caused by mutations in the beta-globin gene, which may cause significantly reduced or completely absent adult hemoglobin (Hb).
Zynteglo (beti-cel) when administered into a transfusion-dependent beta-thalassemia (TDT) patient, restores the functional beta-globin gene into the patient’s hematopoietic (blood) stem cells (HSCs), allowing them to make normal or near normal levels of total hemoglobin without regular RBC transfusions.
The functional beta-globin gene is added to a patient’s cells outside of the body (ex-vivo), and then infused into the patient.
In September 2021. Bluebird completed the submission of its biologics license application (BLA) to the FDA for beti-cel as a treatment for adult, adolescent and pediatric patients with beta-thalassemia who require regular RBC transfusions, across all genotypes.
The BLA was granted priority review by the FDA in November last year.
In June, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted unanimously (13-0) that the benefits of beti-cel gene therapy outweigh the risks for patients with beta-thalassemia who require regular RBC transfusions.
Zynteglo (beti-cel) is now the first and only FDA-approved ex-vivo lentiviral vector gene therapy for TDT patients.
Shares of BLUE are up 6.93% after market hours on Aug 17, after the announcement of the news. However, the stock has declined 32.2% compared to the industry’s decline of 17.1%.
Image Source: Zacks Investment Research
With the FDA approval in place, bluebird also announced its plans to commercialize Zynteglo in the United States, to enable patient access to the drug as soon as possible.
However, due to the complex nature of the gene therapy, presently it will be available exclusively at Qualified Treatment Centers (QTCs), who has the required expertise in the relevant area. The centers will also receive specialized training to administer Zynteglo in patients.
The gene therapy which represents a potential cure for beta-thalassemia will be available at a wholesale acquisition cost set by the company of $2.8 million per patient; the price representing the curative benefit of the therapy.
However, bluebird has said that it would reimburse contracted government and commercial health insurers for as much as 80% of the treatment’s cost if it stops working, up to two years after receiving it.
Immunovant’s loss per share estimates for 2022 have narrowed from $1.66 to $1.64 in the past 30 days. The same for 2023 has narrowed from $1.94 to $1.75 in the same time frame.
Earnings of Immunovant missed estimates in three of the trailing four quarters and beat the same on the remaining one occasion. The average negative earnings surprise for IMVT is 8.97%.
Inozyme Pharma’s loss estimates for 2022 have narrowed to $2.08 from a loss of $2.10 in the past 30 days. The loss estimates for 2023 has remained has also narrowed down from $2.01 per share to $1.95 per share in the same time frame.
INZY surpassed earnings in three the trailing four quarters, missing the same in one. The average earnings surprise for Inozyme is 6.65%.
Sutro Biopharma’s loss per share estimates for 2022 have narrowed from $3.27 to $2.36 in the past 30 days. The same for 2023 has narrowed from $3.43 to $2.91 in the same time frame.
Earnings of Sutro missed estimates in two of the trailing four quarters, beat the same in one occasion and was in line with the estimate in the other remaining occasion. The average negative earnings surprise for STRO is 1.13%.
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bluebird's (BLUE) Gene Therapy Gets FDA Nod for Beta-Thalassemia
bluebird bio (BLUE - Free Report) recently announced that the FDA has approved Zynteglo (betibeglogene autotemcel), as a one-time gene therapy for treating beta-thalassemia, in adult and pediatric patients who require regular red blood cell (RBC) transfusions
Beta-thalassemia is a severe genetic blood disease caused by mutations in the beta-globin gene, which may cause significantly reduced or completely absent adult hemoglobin (Hb).
Zynteglo (beti-cel) when administered into a transfusion-dependent beta-thalassemia (TDT) patient, restores the functional beta-globin gene into the patient’s hematopoietic (blood) stem cells (HSCs), allowing them to make normal or near normal levels of total hemoglobin without regular RBC transfusions.
The functional beta-globin gene is added to a patient’s cells outside of the body (ex-vivo), and then infused into the patient.
In September 2021. Bluebird completed the submission of its biologics license application (BLA) to the FDA for beti-cel as a treatment for adult, adolescent and pediatric patients with beta-thalassemia who require regular RBC transfusions, across all genotypes.
The BLA was granted priority review by the FDA in November last year.
In June, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted unanimously (13-0) that the benefits of beti-cel gene therapy outweigh the risks for patients with beta-thalassemia who require regular RBC transfusions.
Zynteglo (beti-cel) is now the first and only FDA-approved ex-vivo lentiviral vector gene therapy for TDT patients.
Shares of BLUE are up 6.93% after market hours on Aug 17, after the announcement of the news. However, the stock has declined 32.2% compared to the industry’s decline of 17.1%.
Image Source: Zacks Investment Research
With the FDA approval in place, bluebird also announced its plans to commercialize Zynteglo in the United States, to enable patient access to the drug as soon as possible.
However, due to the complex nature of the gene therapy, presently it will be available exclusively at Qualified Treatment Centers (QTCs), who has the required expertise in the relevant area. The centers will also receive specialized training to administer Zynteglo in patients.
The gene therapy which represents a potential cure for beta-thalassemia will be available at a wholesale acquisition cost set by the company of $2.8 million per patient; the price representing the curative benefit of the therapy.
However, bluebird has said that it would reimburse contracted government and commercial health insurers for as much as 80% of the treatment’s cost if it stops working, up to two years after receiving it.
bluebird bio, Inc. Price
bluebird bio, Inc. price | bluebird bio, Inc. Quote
Zacks Rank and Stocks to Consider
bluebird currently has a Zacks Rank #3 (Hold).
Some better-ranked stocks in the same sector includeImmunovant (IMVT - Free Report) , Inozyme Pharma (INZY - Free Report) and Sutro Biopharma (STRO - Free Report) , each carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 (Strong Buy) Rank stocks here.
Immunovant’s loss per share estimates for 2022 have narrowed from $1.66 to $1.64 in the past 30 days. The same for 2023 has narrowed from $1.94 to $1.75 in the same time frame.
Earnings of Immunovant missed estimates in three of the trailing four quarters and beat the same on the remaining one occasion. The average negative earnings surprise for IMVT is 8.97%.
Inozyme Pharma’s loss estimates for 2022 have narrowed to $2.08 from a loss of $2.10 in the past 30 days. The loss estimates for 2023 has remained has also narrowed down from $2.01 per share to $1.95 per share in the same time frame.
INZY surpassed earnings in three the trailing four quarters, missing the same in one. The average earnings surprise for Inozyme is 6.65%.
Sutro Biopharma’s loss per share estimates for 2022 have narrowed from $3.27 to $2.36 in the past 30 days. The same for 2023 has narrowed from $3.43 to $2.91 in the same time frame.
Earnings of Sutro missed estimates in two of the trailing four quarters, beat the same in one occasion and was in line with the estimate in the other remaining occasion. The average negative earnings surprise for STRO is 1.13%.