According to a recent press release from Bluebird Bio, the European Medicines Agency has accepted and will review their marketing authorization application (MAA) for the investigational gene therapy LentiGlobin™ as a treatment for adolescents and adults who have transfusion-dependent β-thalassemia and a non-β0/β0 genotype. To view Bluebird Bio’s original press release, click here.
Thalassemia is a group of genetic blood disorders that decrease the amount of haemoglobin the body produces. Haemoglobin is a protein that is used in red blood cells, which carry oxygen around the body. β-thalassemia is a sub-category of thalassemia, and there are multiple types of β-thalassemia. Since people with β-thalassemia have low levels of haemoglobin, their red blood cells may not be able to transport enough oxygen to parts of the body. Low levels of red blood cells can also make people with the disorder anaemic, which may cause them to be tired, short of breath, and pale. Different types of β-thalassemia are associated with different symptoms, and the disorder has a spectrum of severity.
According to NIH, β-thalassemia is caused by mutations in the HBB gene that codes for the beta-globin protein, which is a component of haemoglobin. Depending on their specific mutation, some people may not be able to produce any beta-globin, and this is called B0 thalassemia. Other mutations may be able to create some beta-globin, but only in small amounts. This is called B+ thalassemia. However, knowing which mutation a person has doesn’t always predict how severe their disorder will be. For more information about the genetic causes underlying β-thalassemia, you can visit the NIH’s website here.
About LentiGlobinTM Gene Therapy
LentiGlobin is an investigational gene therapy that is being developed to treat the genetic cause of thalassemia in patients with transfusion-dependent β-thalassemia (TDT) and non-β0/β0 genotype. According to Bluebird, people with TDT have a lifelong dependence on blood transfusions, which can lead to iron overload. LentiGlobin is designed to be a one-time therapy that reduces the need for patients with TDT to undergo blood transfusions. Bluebird says that by decreasing the number of blood transfusions patients need, the long-term complications of TDT could also be reduced.
Research into LentiGlobin
LentiGlobin is being investigated in multiple studies taking place around the world, including in Australia, Europe, Thailand, and the US. Bluebird is also holding a long-term follow-up study for people who took part in other trials of LentiGlobin for TDT and for sickle cell disease, which will provide more evidence about the therapy’s long-term safety.
The Marketing Authorisation Application
The European Medicines Agency (EMA) is evaluating a marketing authorisation application for LentiGlobin as a treatment for TDT. If approved, this means that LentiGlobin could be marketed in the European Union. EMA will look at Bluebird’s application under Accelerated Assessment, which means that the review period could be shorted from 210 days to 150. To find out more about marketing authorisation, click here.