Zyntelgo has approval in Europe
Treats severe transfusion-dependent beta-thalassemia. Still waiting on FDA.December 11, 2019
Drug name: Zyntelgo® (betibeglogene darolentivec)
Manufacturer: Bluebird Bio
Condition: Transfusion-dependent beta-thalassemia (TDT)
Beta-thalassemia is a severe genetic blood disorder that makes it difficult for red blood cells to carry oxygen.¹ It causes mutations in the gene that makes beta-globin protein. Beta-globin is a component of hemoglobin, the red blood cells that carry oxygen to the whole body. Beta-thalassemia affects one in 100,000 people worldwide.² The most severe cases require regular blood transfusions. This is referred to as transfusion-dependent beta-thalassemia (TDT).
Symptoms depend on the severity of the condition and are generally noted during childhood or adolescence. Common symptoms can include anemia, fatigue and iron overload, which can lead to multiple organ failure.
There is no known cure for TDT. The most severe cases require regular blood transfusions, which unfortunately can also cause a toxic buildup of iron in the blood.
Zynteglo™ received orphan drug and breakthrough therapy designations from the U.S. Food and Drug Administration (FDA).² Zynteglo, a one-time gene replacement therapy, has three Phase 3 studies currently underway. Zynteglo targets the main cause of TDT by restoring the beta-globin protein in the blood. It does this by adding normal copies of the modified beta-globin gene through hematopoietic stem cell transplantation.²
In early June 2019, Bluebird Bio received approval from the European Medicines Agency (EMA) for Zynteglo for patients ages 12 years and over with TDT. In Europe, Bluebird set the price at $1.8 million, and proposed an installment plan of $350,000/year, noting that if the drug failed during the first year, the four remaining payments would not be necessary.
Bluebird expects the therapy to be approved in the U.S. in 2020.¹
PDFUA: TBD (2020)
Benefit: Zyntelgo™ is gene therapy and will impact the medical benefit.
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