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FDA Committee Endorses bluebird's (BLUE) Thalassemia Therapy
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bluebird bio (BLUE - Free Report) announced that the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) has unanimously recommended approval of its gene therapy candidate betibeglogene autotemcel (beti-cel) to treat beta-thalassemia, a severe genetic blood disorder.
The CTGTAC voted 13-0, unanimously recommending the benefits of treatment with beti-cel to outweigh the risks.
bluebird bio’s biologics license application (BLA) filed with the FDA seeks approval for beti-cel to treat patients with beta-thalassemia who require regular red blood cell (RBC) transfusions. The filing was granted a priority review by the FDA last November and the regulatory agency’s final decision on the gene therapy is expected by Aug 19, 2022.
Shares of bluebird bio have plunged 62.7% in the year so far compared with the industry’s 27.5% decline.
Image Source: Zacks Investment Research
The CTGTAC’s positive recommendation was based on data from multiple clinical studies, namely the ongoing phase III HGB-207 and HGB-212 studies, and the completed phase I/II HGB-204 and HGB-205 studies that evaluate beti-cel in beta thalassemia patients across all ages and genotypes.
Based on the late-stage clinical studies conducted by bluebird bio on beti-cel, 89% of evaluable patients achieved transfusion independence, which is defined as no longer needing RBC transfusions for at least 12 months while maintaining an average hemoglobin (hb) of at least 9g/dL.
Beti-cel involves adding functional copies of a modified form of the beta-globin gene into thalassemia patients’ own hematopoietic (blood) stem cells to enable the formation of normal RBCs in patients, without the need for transfusions.
Since last week, some good news has been driving BLUE’s prospects. Apart from the above recommendation for beti-cel, the CTGTAC unanimously recommended approval for another bluebird bio’s regulatory filing, which seeks approval for elivaldogene autotemcel (eli-cel) to treat cerebral adrenoleukodystrophy (CALD), a rare neurodegenerative disease.The CTGTAC voted 15-0, recommending the benefits of treatment with eli-cel to offset the risks. A final decision from the FDA is expected by Sep 16, 2022.
bluebird bio is currently facing the risk of a severe cash crunch. In fact, management expressed concerns over BLUE’s capacity to continue as a going concern due to the pressure on its existing cash balance, which is expected to dry up in the near future.
Presently, BLUE has no approved/marketed drug in its portfolio. A tentative approval for either beti-cel or eli-cel will provide bluebird bio with its first FDA-approved drug.
An approval of either or both beti-cel and eli-cel will also make bluebird bio eligible for priority review vouchers (PRVs) from the FDA. BLUE anticipates selling these vouchers to boost its cash resources.
bluebird bio carries a Zacks Rank #3 (Hold) at present. Some better-ranked stocks in the overall healthcare sector are Abeona Therapeutics , Alkermes (ALKS - Free Report) and Sesen Bio . While both Alkermes and Sesen Bio sport a Zacks Rank #1 (Strong Buy) at present, Abeona Therapeutics carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Alkermes’ loss per share estimates for 2022 have narrowed from 14 cents to 3 cents in the past 60 days. Shares of ALKS have risen 18.1% year to date.
Earnings of Alkermes beat estimates in each of the last four quarters, the average being 350.5%. In the last reported quarter, ALKS delivered an earnings surprise of 1,100%.
Sesen Bio’s loss per share estimates for 2022 have declined from 33 cents to 32 cents in the past 60 days. Shares of SESN have fallen 10.5% in the year-to-date period.
Earnings of Sesen Bio beat estimates in three of the last four quarters and missed the mark on one occasion, the average surprise being 69.9%. In the last reported quarter, SESN delivered an earnings surprise of 100%.
Abeona Therapeutics’ loss per share estimates for 2022 have narrowed from 34 cents to 31 cents in the past 30 days. Shares of ABEO have plunged 49.6% in the year-to-date period.
Earnings of Abeona Therapeutics missed estimates in two of the trailing four quarters and met the same on the remaining two occasions, the average negative surprise being 8.2%. In the last reported quarter, ABEO missed on earnings by 25%.
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FDA Committee Endorses bluebird's (BLUE) Thalassemia Therapy
bluebird bio (BLUE - Free Report) announced that the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) has unanimously recommended approval of its gene therapy candidate betibeglogene autotemcel (beti-cel) to treat beta-thalassemia, a severe genetic blood disorder.
The CTGTAC voted 13-0, unanimously recommending the benefits of treatment with beti-cel to outweigh the risks.
bluebird bio’s biologics license application (BLA) filed with the FDA seeks approval for beti-cel to treat patients with beta-thalassemia who require regular red blood cell (RBC) transfusions. The filing was granted a priority review by the FDA last November and the regulatory agency’s final decision on the gene therapy is expected by Aug 19, 2022.
Shares of bluebird bio have plunged 62.7% in the year so far compared with the industry’s 27.5% decline.
Image Source: Zacks Investment Research
The CTGTAC’s positive recommendation was based on data from multiple clinical studies, namely the ongoing phase III HGB-207 and HGB-212 studies, and the completed phase I/II HGB-204 and HGB-205 studies that evaluate beti-cel in beta thalassemia patients across all ages and genotypes.
Based on the late-stage clinical studies conducted by bluebird bio on beti-cel, 89% of evaluable patients achieved transfusion independence, which is defined as no longer needing RBC transfusions for at least 12 months while maintaining an average hemoglobin (hb) of at least 9g/dL.
Beti-cel involves adding functional copies of a modified form of the beta-globin gene into thalassemia patients’ own hematopoietic (blood) stem cells to enable the formation of normal RBCs in patients, without the need for transfusions.
Since last week, some good news has been driving BLUE’s prospects. Apart from the above recommendation for beti-cel, the CTGTAC unanimously recommended approval for another bluebird bio’s regulatory filing, which seeks approval for elivaldogene autotemcel (eli-cel) to treat cerebral adrenoleukodystrophy (CALD), a rare neurodegenerative disease.The CTGTAC voted 15-0, recommending the benefits of treatment with eli-cel to offset the risks. A final decision from the FDA is expected by Sep 16, 2022.
bluebird bio is currently facing the risk of a severe cash crunch. In fact, management expressed concerns over BLUE’s capacity to continue as a going concern due to the pressure on its existing cash balance, which is expected to dry up in the near future.
Presently, BLUE has no approved/marketed drug in its portfolio. A tentative approval for either beti-cel or eli-cel will provide bluebird bio with its first FDA-approved drug.
An approval of either or both beti-cel and eli-cel will also make bluebird bio eligible for priority review vouchers (PRVs) from the FDA. BLUE anticipates selling these vouchers to boost its cash resources.
bluebird bio, Inc. Price
bluebird bio, Inc. price | bluebird bio, Inc. Quote
Zacks Rank & Stocks to Consider
bluebird bio carries a Zacks Rank #3 (Hold) at present. Some better-ranked stocks in the overall healthcare sector are Abeona Therapeutics , Alkermes (ALKS - Free Report) and Sesen Bio . While both Alkermes and Sesen Bio sport a Zacks Rank #1 (Strong Buy) at present, Abeona Therapeutics carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Alkermes’ loss per share estimates for 2022 have narrowed from 14 cents to 3 cents in the past 60 days. Shares of ALKS have risen 18.1% year to date.
Earnings of Alkermes beat estimates in each of the last four quarters, the average being 350.5%. In the last reported quarter, ALKS delivered an earnings surprise of 1,100%.
Sesen Bio’s loss per share estimates for 2022 have declined from 33 cents to 32 cents in the past 60 days. Shares of SESN have fallen 10.5% in the year-to-date period.
Earnings of Sesen Bio beat estimates in three of the last four quarters and missed the mark on one occasion, the average surprise being 69.9%. In the last reported quarter, SESN delivered an earnings surprise of 100%.
Abeona Therapeutics’ loss per share estimates for 2022 have narrowed from 34 cents to 31 cents in the past 30 days. Shares of ABEO have plunged 49.6% in the year-to-date period.
Earnings of Abeona Therapeutics missed estimates in two of the trailing four quarters and met the same on the remaining two occasions, the average negative surprise being 8.2%. In the last reported quarter, ABEO missed on earnings by 25%.