What is Zynteglo (Betibeglogene Autotemcel)?
Betibeglogene Autotemcel, brand name Zynteglo, is a one-time gene therapy used to treat transfusion-dependent beta-thalassemia. It works by modifying a patient’s own blood-forming stem cells with a functional beta-globin gene, delivered via a viral vector. A viral vector is a modified virus used as a tool to deliver genetic material into a target cell.
This modified virus “hijacks” the virus’s natural ability to enter cells, but with the disease-causing parts of the virus removed and replaced with the desired genetic material. Viral vectors are used in gene therapy to correct or replace faulty genes and in vaccine development to stimulate an immune response.
Once delivered, the betibeglogene autotemcel allows the body to produce healthy red blood cells, avoiding the need for regular blood transfusions. A patient’s stem cells are gathered, genetically modified in a lab to include a functional copy of the beta-globin gene, then infused back into the body following a myeloablative procedure to clear the bone marrow.
The specific viral vector used in this procedure is known as a lentiviral vector, based on a modified version of HIV. Once infused back into the bloodstream, the modified cells graft into the bone marrow and begin producing healthy red blood cells.
What Illnesses Can Benefit from Zynteglo?
Zynteglo is approved for eligible children and adults with beta-thalassemia major, who require regular red blood cell transfusions. The entire process can take several months, from start to finish. There are certain risks involved with Zynteglo, and in some cases, a false-positive HIVE test result could occur. Clinical trials have shown that Zynteglo can allow many patients to achieve transfusion independence.
When Did the FDA Approve Zynteglo and What is the Cost?
The FDA approved betibeglogene autotemcel on August 17, 2022, as the first cell-based gene therapy for pediatric and adult patients with transfusion-dependent beta thalassemia. Beta thalassemia is an inherited disease caused by mutations in the beta-globin gene. Patients with the most severe form of the disease require lifelong red blood cell transfusions, which can lead to chronic iron overload and organ toxicity.
The only other approved treatment for patients with beta-thalassemia is stem cell transplantation, which is limited by donor availability and graft-versus-host disease. In the Phase III study of Zynteglo, 89% of patients receiving the drug achieved transfusion independence. The price of Zynteglo is $2.8 million per treatment, making it one of the most expensive drugs in the world. The high cost is due to the complex manufacturing process and the fact that Zynteglo is a one-time treatment that can potentially cure beta thalassemia.
Why Might You Receive a Zynteglo Gene Therapy Treatment Denial?
Receiving a denial from your insurer for Zynteglo is actually more likely than not. Insurance denials can be issued for a variety of reasons. Some of these reasons are easily fixed, such as incomplete information or a miscoding of a treatment. When you receive a denial letter from your insurer, it must state why the claim was denied and provide you with information for the next steps.
Insurance companies may deny a treatment because it is out-of-network, or because the plan documents specifically state that this treatment is not covered. More often, a treatment is denied as being “not medically necessary,” “experimental,” or “investigational.” These words are often meant to hide the fact that the drug, treatment, or procedure is expensive, and the insurance company does not want to pay. If your betibeglogene autotemcel claim has been denied, this does not have to be the end.
What Should You Do if Your Zynteglo Therapy is Denied by Insurance?
If your Zynteglo gene therapy has been denied by insurance, read the denial letter carefully to see why it was denied. If it is something that can be easily fixed, do so, and resubmit the claim. If it has been deemed not medically necessary, experimental, or investigational, there is an appeal process. You will first file an internal appeal, essentially asking your insurance company to reconsider its decision.
It can be helpful to include a letter from your physician, explaining why this treatment is necessary for you and how it will help your short and long-term health. If the insurer still denies the treatment, you can file an external appeal, which goes to a neutral third party. The decision from the external appeal is binding on your insurer. For this reason, we suggest you speak with a highly experienced health insurance denial attorney prior to filing an external appeal (also known as an Independent Medical Review, or IMR in California).
It is understandable that when you are not feeling well, the appeal process can seem overwhelming. You have a strong legal advocate in attorney Scott Glovsky and his legal team. We help people just like you every day and will work hard to ensure you get the medical treatments your doctor feels are necessary for your health and your future.
Which Specific Insurance Companies Are Known to Deny Gene Cell Therapies?
Zynteglo can be denied by virtually any insurer, but could be more likely with some insurers than others. Insurers like Blue Shield of California have medical policies that require clinical review and prior authorization for all Zynteglo requests. Coverage may be provided for FDA-approved indications when medical necessity is documented. California insurers are likely to try to dodge paying for Zynteglo whenever possible because of the cost. Insurers in California who may deny Zynteglo and other necessary medical treatments include:
How the Law Offices of Scott Glovsky Can Help with Zynteglo Gene Therapy Denied by Insurance?
If you or a loved one has had your claim for Zynteglo denied by your insurance company, your first call should be to the Law Offices of Scott Glovsky. Scott will fight for you to get the treatment you need for your health. Like most people, you probably pay your health insurance premiums faithfully and do everything you are supposed to do.
In return, you expect your insurer to protect you and your family when you need it most. Unfortunately, many insurance companies fall short in providing the support you need and deserve. Scott Glovsky and his highly experienced legal team will work to get your treatment approved by your insurer while you have the time you need to care for your health. Contact the Law Offices of Scott Glovsky today.