Bluebird's Zynteglo Gets EU Conditional Marketing Approval

bluebird bio, Inc. (NASDAQ:BLUE) announced that the European Commission (EC) has granted conditional marketing authorization to Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene), the company’s first gene therapy, for patients aged 12 years or older with transfusion-dependent β-thalassemia (TDT), who do not have a β0/β0 genotype and for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

TDT is a severe genetic disease caused by mutations in the β-globin gene that result in reduced or absent hemoglobin.

Shares of the company increased 20% year to date against the industry’s decline of 0.5%.

Zynteglo was reviewed as part of the European Medicines Agency’s (EMA) Priority Medicines (PRIME) and Adaptive Pathways programs. These programs support the medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options.

The conditional marketing authorization is supported by efficacy and durability data from the phase I/II HGB-205 study and the completed phase I/II Northstar (HGB-204) study along with available data from the ongoing phase III Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies, and the long-term follow-up LTF-303 study, as of the data cut off on Dec 13, 2018.

Data from phase I/II HGB-205 showed that 75% of patients, who do not have a β⁰/β⁰ genotype, achieved transfusion independence, while in the phase I/II Northstar study, 80% achieved the same. In Northstar-2 study, 80% patients achieved transfusion independence.

The approval makes available a gene therapy for certain patients with TDT that has the potential to transform lives by offering the possibility of a transfusion-free future.

The approval will boost sales for the company.

Zacks Rank and Other Stocks to Consider

bluebird currently has a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are Anika Therapeutics Inc. (NASDAQ:ANIK) , Applied Genetics Technologies Corp. (NASDAQ:AGTC) and Acorda Therapeutics Inc. (NASDAQ:AXGT) . All of them carry a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Anika’s earnings per share estimates have moved up from $1.22 to $1.28 for 2019 and from $1.21 to $1.33 for 2020 in the past 60 days. The company delivered a positive earnings surprise in the trailing four quarters, with average beat of 72.00%.

Applied Genetics’ loss per share estimates have narrowed from $1.25 to 1 cent for 2019 and from $2.39 to $2.15 for 2020 in the past 60 days. The company delivered a positive earnings surprise in three of the trailing four quarters, with average beat of 83.47%.

Acorda’s loss per share estimates have narrowed from $3.84 to $3.59 for 2019 and from $3.32 to $3.09 for 2020 in the past 60 days. The company delivered a positive earnings surprise in all of the trailing four quarters, with average beat of 79.32%.

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