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bluebird's MAA for LentiGlobin Gene Therapy Accepted by EMA

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bluebird bio, Inc. (BLUE - Free Report) announced that the European Medicines Agency (EMA) accepted the marketing authorization application (MAA) for its experimental gene therapy, LentiGlobin, for the treatment of adolescents and adults with transfusion-dependent β-thalassemia (TDT) and a non-β0/β0 genotype.

The MAA is supported by data from the phase I/II Northstar (HGB-204) study and the ongoing phase I/II2 HGB-205 study. The available data from the phase III Northstar-2 (HGB-207) study and the long-term follow-up study LTF-303 also supported the MAA.

The EMA will evaluate the MAA under accelerated assessment, as the Committee for Medicinal Products for Human Use (CHMP) of the EMA had granted the same in July 2018. Under an accelerated assessment procedure, the EMA’s active review time of the MAA is potentially reduced from 210 days to 150 days.

We remind investors that the EMA previously granted Priority Medicines (PRIME) eligibility and Orphan Medicinal Product designation to LentiGlobin for the treatment of TDT.

LentiGlobin is a one-time gene therapy being studied as a potential treatment to address the underlying genetic cause of TDT, which, in turn, might eliminate or reduce the need for blood transfusions.

The FDA had previously granted Orphan Drug status and Breakthrough Therapy designation to the therapy for the treatment of TDT.

Apart from the ongoing studies in various countries, bluebird is conducting a long-term safety and efficacy follow-up study (LTF-303) for patients who have participated in bluebird-sponsored clinical studies of LentiGlobin for TDT and sickle cell disease.

bluebird has built a pipeline with broad potential application in severe genetic diseases and cancer with its lentiviral-based gene therapies, T-cell immunotherapy expertise and gene editing capabilities.

Year to date, shares of bluebird have decreased 19.5% compared with the industry’s decline of 9.7%.

 

Last month, bluebird announced updated results from phase II/III Starbeam study (ALD-102) of its investigational Lenti-D gene therapy in boys aged 17 years or below with cerebral adrenoleukodystrophy (CALD). The company also announced initial results from the ongoing observational ALD-103 study of outcomes from allogeneic hematopoietic stem cell transplant (allo-HSCT) in boys aged 17 years or below with CALD.

The oncology pipeline includes CAR-T cell product candidates — bb2121 and bb21217 — for the treatment of multiple myeloma. The company is co-developing and co-promoting bb2121 in the United States with Celgene Corporation (CELG - Free Report) .

Zacks Rank & Stocks to Consider

bluebird is a Zacks Rank #4 (Sell) stock.

Some better-ranked stocks from the pharma space are Caladrius Biosciences, Inc. (CLBS - Free Report) and Ligand Pharmaceuticals (LGND - Free Report) . While Ligand carries a Zacks Rank #1 (Strong Buy), Caladrius is a Zacks Rank #2 (Buy) stock. You can see the complete list of today’s Zacks #1 Rank stocks here.

Ligand’s earnings per share estimates have moved up from $5.64 to $6.33 for 2018 and from $5.59 to $5.64 for 2019 over the past 60 days. The company delivered a positive earnings surprise in all the trailing four quarters with an average beat of 59.5%.

Caladrius’ loss per share estimates have narrowed from $2.30 to $2.17 for 2018 and from $2.43 to $2.39 for 2019 over the past 60 days. The company delivered a positive earnings surprise in all the trailing four quarters with an average beat of 41.5%.

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