Gene therapy for blood disorders developed by Bluebird Bio has already left the nest in the US.
Zynteglo, sometimes known as betibeglogene autotemcel (beti-cel), was authorised by the FDA on August 17, two days before the FDA’s scheduled approval action date of August 19. The approval is momentous for patients as well as business, according to Tom Klima, Bluebird’s chief commercial officer. Bluebird has experienced its fair share of bumps and bruises over the last year and a half or so, but since a fruitful FDA advisory meeting held earlier this summer, things have been looking brighter, according to Klima.
When an FDA expert panel unanimously backed the gene therapy in June, the uncertainty around bluebird’s chances for approval that had existed at the beginning of the year began to lift. One participant in the discussion asserted that the medication might be life-changing for patients who are now dependent on blood transfusions.
The FDA stated in a release that the approval allows the medicine to be used by children and adults with beta thalassemia who need routine red blood cell transfusions. As a result of the genetic blood condition beta thalassemia, red blood cells and normal haemoglobin levels in the blood are decreased as a result. Insufficient oxygen supply in the body results from this.
The FDA noted that in addition to more serious problems, the disease can result in bone abnormalities, weakness, weariness, and disorientation. The more extreme type of the disorder, transfusion-dependent beta thalassemia, often requires patients to need red blood cell transfusions for the rest of their lives. Transfusions can cause their own set of health issues, such as issues with the heart, liver, and other organs because of an excessive build-up of the body’s iron levels.
Bluebird is to open its first wave of accredited treatment facilities by September, according to Klima. It anticipates beginning the collection of patient cells by the fourth quarter of 2022 with the goal of starting the first patient’s treatment in early 2023, he adds.
Each Zynteglo dose is produced as a one-time customised treatment by genetically altering the patient’s own bone marrow stem cells. The patient’s cells are specifically altered to create functioning beta-globin, a substance found in haemoglobin.
The FDA reported that 89% of the 41 patients taking Zynteglo in two clinical investigations, including adults and children with transfusion-dependent beta thalassemia, attained transfusion independence.
Bluebird’s medicine won’t be cheap, though. Klima said that Zynteglo will launch at a cost of $2.8 million per patient, adding that the business believes the price reflects the worth of a one-time treatment in the U.S. The cost of hospitalisation necessary for patients getting the therapy is not included in the quoted price.
As Bluebird rejoices at the clearance, insurers are undoubtedly paying attention. Bluebird will need to carry out what it couldn’t do in Europe while introducing the medication in the United States: Persuade the payers of Zynteglo’s worth.
According to Klima, Bluebird is now finalising partnerships with commercial payers. As stated by the CCO, the company’s access approach is based on a one-time upfront payment with the potential for a discount for patients who don’t attain transfusion independence. This might allow patients to recover up to 80% of the one-time medication costs.
Zynteglo received clearance from the European Union in 2019 at a cost of about 1.58 million euros (around $1.8 million at the time). However, the business later decided to remove the medication from sale because it was unable to negotiate reimbursement terms with authorities in several areas. Apparently, Bluebird reduced 30% of their workforce in April.
With the U.S. approval, Zynteglo is now considering a market of around 1,000 to 1,500 patients with transfusion-dependent beta thalassemia.
