Home / Business News / Bluebird Down 5% on $1.8 Million Price Tag for Gene Therapy Zynteglo


Bluebird Bio (BLUE) shares were down 5% on Friday afternoon after the company earlier in the day set a price of EUR1.6 million ($1.8 million) over five years for its gene therapy Zynteglo, making it the second most expensive therapy in the world behind Novartis’ (NVS) Zolgensma at $2.1 million, according to media reports.

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A few hours later, Cowen analyst Yaron Werber reported “highly encouraging” Zynteglo data at the European Hematology Association (EHA) meeting, but said that the European Medicines Agency (EMA) has asked for changes, which will cause a delay in the regional launch, Endpoints News reported.

“(T)he key unexpected news is that EMA has requested amendments to the final drug products specifications and to the manufacturing parameters. Hence this delays the launch and would remove any sales in FY19 to early ’20,” Werber reportedly said.

“The good news is that it would enable blue to onboard the qualified treatment centers and help further prep for the launch. However, unquestionably this request adds an element of surprise and would require blue to make a few mnfg process changes ahead of launch which would also add some new risk.”

Zynteglo received conditional approval in Europe this month for patients aged 12 years and older with beta thalassemia who require regular blood transfusions for disease management and have no matching donor for a stem cell transplant.

Bluebird proposed a five-year installment plan for Zynteglo, with 315,000 euros paid up front and additional payments only due if the treatment continues to be effective.

Bluebird, which is also testing Zynteglo for sickle cell disease, expects the therapy to receive approval for beta thalassemia in the US in 2020.

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