BioPharma

bluebird working to open European gene therapy sites, but high price could be a hindrance

An analyst wrote that bluebird bio would likely launch Zynteglo first in Germany, followed by other countries, but scaled back sales forecasts due to the therapy's high price tag of 1.575 million euros.

A biotech company that won the first European approval for a gene therapy to treat the blood disorder beta-thalassemia is busy getting its treatment sites online, and an analyst anticipates that Germany will likely be the first country where it becomes available. However, its high price tag could hinder sales.

Cambridge, Massachusetts-based bluebird bio announced its second quarter earnings Thursday, providing updates on the gene therapy, Zynteglo (autologous CD34-positive cells encoding beta-A-T87Q-globin gene) and bb2121 (idecabtagene vicleucel), a CAR-T for multiple myeloma, among other programs. Zynteglo won European Medicines Agency approval for beta-thalassemia in June.

In a statement, bluebird CEO Nick Leschly said the company is focused on getting treatment centers offering Zynteglo in Europe up and running.

“In the weeks since announcing our approval, we have been encouraged by our progress across these fronts and by the receptivity of our payment model from payers in our initial European launch markets,” Leschly said.

But the price the company announced in the middle of last month, 1.575 million euros ($1.7 million at current exchange rates), was higher than expected, wrote Cowen analyst Yaron Werber in a note to investors Thursday evening. The investment bank had been anticipating it would cost about $750,000.

Based on the higher price, Cowen lowered its sales estimates, projecting that sales would grow from $19 million to $164 million after launch.

Werber wrote that it is likely bluebird will launch Zynteglo first in Germany in early 2020, followed by Italy, France, the U.K. and other countries in the European Union. A rolling regulatory submission to the Food and Drug Administration will start by the end of 2019, with the U.S. launch happening in late 2020.

Zynteglo is being sold on a five-year installment plan, similar to the one that Novartis is using for Zolgensma (onasemnogene abeparvovec-xioi), which the FDA approved in May for spinal muscular atrophy and which Novartis priced at about $2 million.

With respect to bb2121, Werber reiterated from an earlier note on the earnings report of Celgene, with which bluebird is partnered on the CAR-T program, that full data may not be ready in time for the American Society of Hematology meeting in December, but that an FDA filing is likely to take place in early 2020.

Shares of bluebird were down more than 6 percent on the Nasdaq late Friday morning and at midday.

Photo: harmpeti, Getty Images

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