Gene/Cell Therapy Quarterly Update: SEPTEMBER 2022

This quarterly pipeline wrap-up provides a review of newly approved gene and cell therapies, new indications and news of note on gene and cell therapies drugs in the approval process. See other articles for updates on specialty drugs, biosimilar drugs and  traditional drugs. 

New Drug Information

• Zynteglo^®(betibeglogene autotemcel): The FDA has approved Bluebird bio’s Zynteglo also known as beti-cel as a one-time gene therapy to treat the underlying genetic cause of beta‑thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions. Beta-thalassemia, a type of inherited blood disorder, reduces the levels of normal hemoglobin and red blood cells via mutations in the beta-globin subunit; this can ultimately cause the delivery of oxygen throughout the body to be at insufficient levels. The subsequently low levels of red blood cells can result in a number of health problems, such as dizziness, weakness, fatigue, bone abnormalities, and other more serious complications. Zynteglo established the efficacy based on the achievement of transfusion independence, which is the point at which the patient maintains a pre-determined level of hemoglobin without needing any red blood cell transfusions for at least 12 months. Across two clinical studies evaluating adults and kids with transfusion-dependent beta thalassemia, 89% of the 41 patients on Zynteglo achieved transfusion independence.¹ Bluebird bio anticipates its launch in 2023 with a one-time price of $2.8 million per patient.² The list price doesn’t include the cost of hospitalization required for patients receiving the therapy.
• Skysona® (elivaldogene autotemcel): The FDA approved bluebird bio’s Skysona (elivaldogene autotemcel) to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD).  Skysona is a one-time gene therapy that adds functional copies of the ABCD1 gene into a patient’s own hematopoietic stem cells. The accelerated approval was based on the 24-month Major Functional Disabilities (MFDs) in Phase 2/3 study ALD-102 and Phase 3 ALD-104.  Patients treated with Skysona had a 72% likelihood of MFD-free survival at 24 compared to untreated patients who had only a 43% likeliood of MFD-free survival.³Another treatment option allogeneic hematopoietic stem cell transplant (allo-HSCT).  Skysona will be available by the end of 2022 through a limited number of Qualified Treatment Centers in the US at a one-time wholesale acquisition cost of $3 million.

 New Indications

• None