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AVLT and UKTS Announce the European Medicines Agency’s Approval of the Conditional Marketing Authorization Application for Zynteglo™ (Previously Known as Lentiglobin™) Gene Therapy for the Treatment of Transfusion Dependent Beta Thalassaemia

March 25, 2019

LONDON--(BUSINESS WIRE)--Mar 25, 2019--Associazione Veneta Lotta alla Talassemia (AVLT) and the UK Thalassaemia Society (UKTS) announced today that the European Medicines Agency (EMA) has approved bluebird bio’s application for Conditional Marketing Authorization (cMAA) of their product Zynteglo™, a gene therapy for the treatment of adolescents and adults with transfusion-dependent β-thalassaemia (TDT) who do not have a β 0 /β 0  genotype.

As a result of this marketing authorisation, Zynteglo™ is now approved for use in all European countries covered by the European Medicines Agency.

Zynteglo™ has been given ‘conditional authorisation’. This means that there is more evidence to be submitted, which the company is required to provide under specific obligations. Every year, the EMA will review any new information that becomes available.

“People living with transfusion-dependent β-thalassaemia have a reduced life expectancy, requiring life-long frequent blood transfusions that are life-saving but may lead to complications, including organ failure due to iron overload,” said Dr. Michele Lipucci di Paola, of AVLT, patient representative at EMA.

Dr. Chris Sotirelis, an expert patient at EMA said: “The approval of Zynteglo™ is a breakthrough that offers a unique treatment choice to patients, as the first one-time gene therapy that addresses the underlying genetic cause of transfusion-dependent β-thalassaemia.

The opinion adopted by the CHMP at its March 2019 meeting is an intermediary step for Zynteglo™ to path to reach patients. The CHMP opinion, once confirmed by the European Commission, will result in an EU-wide marketing authorisation. After that decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role/use of this medicine in the context of the national health system of that country.

View source version on businesswire.com:https://www.businesswire.com/news/home/20190325005774/en/

CONTACT: AVLT

Dr. Michele Lipucci di Paola

+39 3382104475

mlipucci@yahoo.com

UKTS

Dr. Chris Sotirelis

+44 7979594067

chris.sotirelis@gmail.com

KEYWORD: UNITED KINGDOM EUROPE

INDUSTRY KEYWORD: HEALTH BIOTECHNOLOGY GENETICS PHARMACEUTICAL RESEARCH SCIENCE

SOURCE: UK Thalassaemia Society

Copyright Business Wire 2019.

PUB: 03/25/2019 03:50 PM/DISC: 03/25/2019 03:50 PM

http://www.businesswire.com/news/home/20190325005774/en