Regulatory Focus™ > News Articles > 2019 > 6 > EU Gets a Leg Up on US With First New Gene Therapy for Blood Disorder

EU Gets a Leg Up on US With First New Gene Therapy for Blood Disorder

Posted 03 June 2019 | By Zachary Brennan 

EU Gets a Leg Up on US With First New Gene Therapy for Blood Disorder

The European Commission on Monday granted conditional marketing authorization for Bluebird Bio’s gene therapy Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene) to treat a rare blood disorder.

Zynteglo addresses the underlying genetic cause of transfusion-dependent beta-thalassemia (TDT), a rare inherited blood condition that causes severe anemia. The therapy, which has so far been evaluated in 11 patients, is intended for patients 12 years and older who need regular blood transfusions to manage their disease and do not have a matching donor for a stem cell transplant.

The gene therapy was granted an accelerated assessment in the EU and was reviewed in just 150 days, cutting as much as two months off the time a typical application takes to make it through the centralized procedure.

The European Medicines Agency said it was “the fastest review time for an ATMP [advanced therapy medicinal product] to date.”

In the US, however, Bluebird plans to take a slower approach and does not expect to submit its biologics license application to the US Food and Drug Administration (FDA) until the end of this year. Still, the therapy has already been awarded orphan drug status and breakthrough therapy designation.

As far as the price for Zynteglo, a Bluebird spokesperson told Focus it will announce the amount and other plans by the end of next week. The company previously discussed a value-based payment model.

Leerink analysts also said they had modeled the launch price of LentiGlobin (the old name for Zynteglo) as $1.2 million in the US and $0.9 million in the EU.

Categories: Regulatory News

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