– Het Europees Geneesmiddelenbureau voor Geneesmiddelen voor Menselijk Gebruik (CHMP) heeft een voorwaardelijke handelsvergunning in de Europese Unie goedgekeurd voor Zynteglo™

LONDON–(BUSINESS WIRE)–Associazione Veneta Lotta alla Talassemia (AVLT) en de UK Thalassemia Society (UKTS) hebben vandaag aangekondigd dat het Europees Geneesmiddelenbureau (EMA) de aanvraag van bluebird bio voor de voorwaardelijke handelsvergunning (cMAA) van hun product Zynteglo™ heeft goedgekeurd. Het betreft een gentherapie voor de behandeling van adolescenten en volwassenen met transfusieafhankelijke β-thalassemie (TDT) die geen β0/β0 genotype hebben.

Als gevolg van deze handelsvergunning is Zynteglo™ nu goedgekeurd voor gebruik in alle Europese landen die onder het Europees Geneesmiddelenbureau vallen.

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

– The European Medicines Agency Human Medicines Committee (CHMP) has approved a Conditional Marketing Authorisation in the European Union for Zynteglo™

LONDON–(BUSINESS WIRE)– 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 β⁰/β⁰ 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/

Contacts

AVLT
Dr. Michele Lipucci di Paola
+39 3382104475
mlipucci@yahoo.com

UKTS
Dr. Chris Sotirelis
+44 7979594067
chris.sotirelis@gmail.com