Two thalassaemia charities have reported that Bluebird Bio’s gene therapy for the disease has been approved in the EU – although that seems to be news to the company.
A statement put out by the UK Thalassaemia Society and Associazione Veneta Lotta alla Talassemia (AVLT) in Italy suggests that the EMA’s Committee for Medicinal Products for Human Use (CHMP) has already granted a conditional marketing approval for “Zynteglo” on the first day of its latest meeting yesterday.
Bluebird Bio has however issued its own statement indicating that “no positive opinion has been issued by the CHMP”, adding that it expects to hear on the status of the gene therapy after the current meeting comes to an end on Thursday (28 March).
The charities’ statement seems to be premature, claiming a conditional marketing authorisation has already been awarded to Zynteglo – which they insist is the new name for the gene therapy although Bluebird continues to use LentiGlobin in its statement.
They say Zynteglo “is now approved for use in all European countries covered by the EMA” for the treatment of adolescents and adults with transfusion-dependent beta-thalassaemia (TDT) who do not have a specific genetic profile, although full approval by the European Commission normally follows a few weeks after a positive opinion is granted by the CHMP.
PMLive has asked the EMA for clarification on the status of the Zynteglo/LentiGlobin review but had not received a response at the time of writing.
Leaving the current confusion aside, assuming the EMA does issue a positive opinion for LentiGlobin after the meeting it will be massive news for European patients with thalassaemia, and that may explain the alacrity with which the UKTS and AVLT released their joint statement.
It would be the first one-off gene therapy to be approved for the inherited rare blood disorder, which is caused by a mutation in the beta-globin gene that impairs red blood cell production and leads to severe anaemia.
Currently patients with thalassaemia are treated with red cell transfusions, but these can lead to iron overload that can in turn result in damage to vital organs unless iron-chelating drugs are administered. Some also get haematopoietic stem cell transplants (HSCT), but this is a risky procedure that carries a risk of death and serious complications.
Dr. Chris Sotirelis of UKTS said 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 beta-thalassaemia.”