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bluebird's MAA for Elivaldogene Autotemcel Accepted by EMA
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bluebird Inc. (BLUE - Free Report) announced that the European Medicines Agency (“EMA”) has accepted the company’s marketing authorization application (“MAA”) for its investigational elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy for the treatment of patients with cerebral adrenoleukodystrophy (CALD). CALD is a fatal neurodegenerative disease primarily affecting young boys.
Shares of the company have declined 39.6% year to date compared with the industry’s decline of 3%.
The MAA was supported by data from a phase II/III Starbeam study, which has completed enrollment, the ongoing phase III ALD-104 study and long-term follow-up study (LTF-304).
bluebird bio is currently enrolling patients for ALD-104 study, which is designed to assess the efficacy and safety of eli-cel after myeloablative conditioning using busulfan and fludarabine in patients with CALD. Further, bluebird bio is conducting a long-term safety and efficacy follow-up LTF-304 study for patients who have been treated with eli-cel for CALD and completed two years of follow-up in bluebird bio-sponsored studies.
Data from these studies conducted in patients with early CALD suggested that eli-cel stabilizes the progression of the disease. If approved, eli-cel would represent the first therapy for CALD that uses a patient’s own hematopoietic stem cells, potentially mitigating the risk of life-threatening immune complications associated with transplant using cells from a donor.
In July 2020, the Committee for Medicinal Products for Human Use of the EMA granted an accelerated assessment to elivaldogene autotemcel for the treatment of CALD. The FDA granted Breakthrough Therapy designation to Lenti-D for the treatment of patients with CALD, which should expedite the development and review of the therapy. Lenti-D also enjoys orphan drug status in the Unites States. It was also granted Rare Pediatric Disease designation by the FDA for the treatment of adrenoleukodystrophy. bluebird bio remains on track to submit the Biologics License Application in the United States in mid-2021.
Emergent’s earnings estimates have increased from $4.03 to $6.61 for 2020 and from $5.07 to $7.62 for 2021 over the past 90 days. Shares of the company have increased 85.8% year to date.
Horizon’s earnings estimates have increased from $2.00 to $2.86 for 2020 and from $3.05 to $4.34 for 2021 over the past 60 days. Shares of the company have increased 120.5% year to date.
Ligand’s earnings estimates have increased from $3.70 to $4.00 for 2020 and from $4.40 to $8.00 for 2021 over the past 60 days. Shares of the company have decreased 6.8% year to date.
5 Stocks Set to Double
Each was hand-picked by a Zacks expert as the #1 favorite stock to gain +100% or more in 2020. Each comes from a different sector and has unique qualities and catalysts that could fuel exceptional growth. Most of the stocks in this report are flying under Wall Street radar, which provides a great opportunity to get in on the ground floor.
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bluebird's MAA for Elivaldogene Autotemcel Accepted by EMA
bluebird Inc. (BLUE - Free Report) announced that the European Medicines Agency (“EMA”) has accepted the company’s marketing authorization application (“MAA”) for its investigational elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy for the treatment of patients with cerebral adrenoleukodystrophy (CALD). CALD is a fatal neurodegenerative disease primarily affecting young boys.
Shares of the company have declined 39.6% year to date compared with the industry’s decline of 3%.
The MAA was supported by data from a phase II/III Starbeam study, which has completed enrollment, the ongoing phase III ALD-104 study and long-term follow-up study (LTF-304).
bluebird bio is currently enrolling patients for ALD-104 study, which is designed to assess the efficacy and safety of eli-cel after myeloablative conditioning using busulfan and fludarabine in patients with CALD. Further, bluebird bio is conducting a long-term safety and efficacy follow-up LTF-304 study for patients who have been treated with eli-cel for CALD and completed two years of follow-up in bluebird bio-sponsored studies.
Data from these studies conducted in patients with early CALD suggested that eli-cel stabilizes the progression of the disease. If approved, eli-cel would represent the first therapy for CALD that uses a patient’s own hematopoietic stem cells, potentially mitigating the risk of life-threatening immune complications associated with transplant using cells from a donor.
In July 2020, the Committee for Medicinal Products for Human Use of the EMA granted an accelerated assessment to elivaldogene autotemcel for the treatment of CALD. The FDA granted Breakthrough Therapy designation to Lenti-D for the treatment of patients with CALD, which should expedite the development and review of the therapy. Lenti-D also enjoys orphan drug status in the Unites States. It was also granted Rare Pediatric Disease designation by the FDA for the treatment of adrenoleukodystrophy. bluebird bio remains on track to submit the Biologics License Application in the United States in mid-2021.
bluebird bio, Inc. Price
bluebird bio, Inc. price | bluebird bio, Inc. Quote
Zacks Rank & Stocks to Consider
bluebird currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the healthcare sector include Emergent Biosolutions Inc. (EBS - Free Report) , Horizon Therapeutics Public Limited Company and Ligand Pharmaceuticals Inc. (LGND - Free Report) , each sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Emergent’s earnings estimates have increased from $4.03 to $6.61 for 2020 and from $5.07 to $7.62 for 2021 over the past 90 days. Shares of the company have increased 85.8% year to date.
Horizon’s earnings estimates have increased from $2.00 to $2.86 for 2020 and from $3.05 to $4.34 for 2021 over the past 60 days. Shares of the company have increased 120.5% year to date.
Ligand’s earnings estimates have increased from $3.70 to $4.00 for 2020 and from $4.40 to $8.00 for 2021 over the past 60 days. Shares of the company have decreased 6.8% year to date.
5 Stocks Set to Double
Each was hand-picked by a Zacks expert as the #1 favorite stock to gain +100% or more in 2020. Each comes from a different sector and has unique qualities and catalysts that could fuel exceptional growth. Most of the stocks in this report are flying under Wall Street radar, which provides a great opportunity to get in on the ground floor.
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