Zyntelgo has approval in Europe

Treats severe transfusion-dependent beta-thalassemia. Still waiting on FDA.

December 11, 2019
At Prime, we get ahead of drug trends to prepare for new, high-cost drugs entering the market. We’ve been following Zyntelgo® (betibeglogene darolentivec) since its early June 2019 approval in Europe.

Drug name: Zyntelgo® (betibeglogene darolentivec)
Manufacturer: Bluebird Bio
Condition: Transfusion-dependent beta-thalassemia (TDT)

Condition overview

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.

Current treatments

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.

Zyntelgo overview

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.

Prime monitors the drug pipeline

The drug pipeline is full of new, groundbreaking specialty drugs that may help members feel better and live well. Prime focuses on clinical strategies designed to keep clients ahead of drug trends — because it’s easier to manage change when you see it coming.


  1. https://www.biospace.com/article/bluebird-bio-presents-more-data-on-its-1-8-million-gene-therapy-zynteglo/
  2. https://www.clinicaltrialsarena.com/projects/zynteglo-beta-thalassemia/

Related news


September 14, 2023

Cheers to 20 years and another amazing Specialty Summit!

The 20th annual Specialty Summit hosted by Prime and Magellan Rx returned to…


September 8, 2023

October 2023 decisions expected from the FDA

At Prime Therapeutics (Prime), we’ve positioned ourselves to best prepare our clients to…


August 28, 2023

Specialty Drug Pipeline Monthly Update: August 2023

This monthly pipeline wrap-up provides a review of newly approved specialty drugs, recent…