Summary
- bluebird bio announced a positive, long-term follow-up of 5 years for beta-thalassemia patients who have been transfusion-free for an extended period of time.
- The rolling BLA submission for ZYNTEGLO to the FDA for transfusion-dependent beta thalassemia patients who do not have the ?0/?0 genotype is expected by end of 2019.
- The first commercial patient to be dosed with ZYNTEGLO for transfusion-dependent beta thalassemia in the European Union is expected in early 2020.
- The cost of ZYNTEGLO is expected to be $1.8 million per patient, but it will be split at 315,000 euros per year over a five-year period and that will be dependent upon the fact that the treatment is actually effective in treating the disease.
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bluebird bio (NASDAQ:BLUE) announced positive results from several studies using its LentiGlobin gene therapy to treat patients with beta-thalassemia. The more prominent news item involves long-term data from one study, which showed that those treated with the therapy were able to achieve transfusion independence for as long as five years. This long-term data, along with several other positive studies, proves that LentiGlobin is a solid treatment option for patients who have this disease. It is imperative that these patients achieve long-term transfusion independence for an improvement of quality of life. In coming weeks, bluebird anticipates the rolling NDA submission of ZYNTEGLO (LentiGlobin) expected for transfusion-dependent beta thalassemia patients (TDT) who do not have the ?0/?0 genotype by the end of 2019. With refined manufacturing specifications for ZYNTEGLO approved by the European medicines Agency (EMA), the biotech expects to treat the first commercial patient by early 2020.
Results Prove LentiGlobin Can Be An Important Treatment Option For Beta-Thalassemia
The reported data is not only important for the advancement of LentiGlobin in the clinic, but also as a more important treatment option for patients with beta-thalassemia. These patients with beta-thalassemia have a major issue when it comes to hemoglobin. They have low levels of hemoglobin in the body, which is bad because oxygen is not able to get to many parts of the body. Hemoglobin is an iron-containing protein in red blood cells. They are important in circulating oxygen throughout the body. However, these patients are in much worse shape from these studies, because they have something known as Transfusion dependent beta-thalassemia (TDT). From a quality of life aspect, these patients have a major burden. Such a burden is the need for constant blood transfusions. Matter of fact, they rely on blood transfusions on a regular basis. What's the problem with that? Besides the hassle of having to do go to an outpatient center to receive a blood transfusion for several hours a time, the patient ends up with something known as iron overload. Iron overload is too much iron that builds up on the organs as a result of regular blood transfusions. This is a major problem because the organs become poisoned which leads to several complications such as:
- An irregular heartbeat
- Cirrhosis of the liver
- Cancer
The use of chronic and or regular blood transfusions is reliant on the hemoglobin levels observed upon examination by a Doctor. A patient with less than 6 g/dL of hemoglobin will definitely be required regular blood transfusions. Positive results were observed in all three studies highlighted by bluebird bio which were Northstar, Northstar-2 and Northstar-3 respectively. All studies treated TDT patients who either did not have a ?0/?0 genotype or who did have a ?0/?0 genotype. Think of those with the ?0/?0 genotype as being more severe in disease in terms of lacking hemoglobin production. They have both genes that are impaired in this regard. That is why this genotype is considered to be a more severe case. Regardless, LentiGlobin performed well in all studies. Patients treated with the company's gene therapy were able to achieve transfusion independence (TI) for long periods of time. Most notably, the long-term follow-up study is highly ideal in this regard. This was the phase 1/2 Northstar (HGB-204) study which had evaluated patients for up to five years or an average of 44.9 months. The patients in this study were able to achieve TI for a long period of time. Not only that, but hemoglobin levels also were normal as well. In order for these patients to be considered transfusion independent, they had to have Hb levels ? 9 g/dL without red blood cell (RBC) transfusions for more than 12 months.
