Zynteglo Approved as First Gene Therapy for Beta-Thalassemia

On August 17, bluebird bio announced that the FDA approved Zynteglo (betibeglogene autotemcel), also known as beti-cel, for the treatment of beta-thalassemia. The drug is intended to be 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.

The cost for Zynteglo will make it one of the most expensive medicines sold at an estimated list price of $2.8 million for the one-time treatment. This upfront price does not include the hospital stay, which is estimated to last 3−6 weeks after infusion. Zynteglo will be available through a network of qualified treatment centers, with the first infusion targeted for Q1 2023.

The Gene Therapy Market

Until mid-August, only two other gene therapy treatments had been approved by the U.S. Food and Drug Administration: Luxturna, which treats a hereditary blindness condition with ingredient cost of $850,000 per patient; and Zolgensma, which treats Spinal Muscular Atrophy (SMA) with an ingredient cost of $2,125,000 per patient. Zynteglo marks the third approval in this rapidly expanding market, which is expected to grow exponentially over the next five to ten years, based on the current drug pipeline.

What Does this Mean for Payers?

From a clinical standpoint, this treatment can result in a tremendous improvement in quality of life for patients. In addition, this curative treatment will potentially eliminate the need for regular, ongoing blood transfusions. However, these lifesaving therapeutics present an enormous financial risk to self-insured plan sponsors, stop loss carriers, payers, and reinsurers. BCS has developed clinical and financial stop-loss and reinsurance solutions designed specifically to help protect against this growing category of expensive and unpredictable high-cost claims, including emerging cell and gene therapies.

For more information about coverage that may be available, contact Mehb Khoja at mkhoja@bcsf.com or Blake Schraft at bschraft@bcsf.com.

Source: IPD Analytics Payer and Provider Update, August 2022