Gene Therapy Drug Insurance Denial Attorney

Lawyers to Help with Gene Therapy Drug Insurance Denials

Gene therapy may truly feel like a lifeline for those with serious diseases, yet when they receive a gene therapy drug insurance denial, that lifeline feels as though it has been jerked away. If you or a loved one have been told by your doctor that gene therapy could offer the hope for treatment and recovery you need, yet your insurance company denies payment for the treatment, attorney Scott Glovsky can help.

Scott understands how discouraging—not to mention potentially damaging to your health—it can be to spend years paying health insurance premiums only to have your legitimate claim denied. At the Law Offices of Scott Glovsky, we fight for your rights so you can receive the medical treatment you need. When your health and your future are at stake, choosing a highly experienced insurance bad faith attorney like Scott Glovsky can make all the difference.

Attorney Scott Glovsky knows every tactic used by insurance companies—delay, deny, underpay. The consequences of these tactics can range from inconvenient to catastrophic. Having Scott on your side after your insurance company has denied your doctor’s request for gene therapy can change your life from disheartening to hopeful once again. If you are in California, call the Law Offices of Scott Glovsky immediately. We have offices in Los Angeles and help residents across the state of California.

What is Gene Therapy?

Medical techniques that use genes to treat or prevent disease are known as gene therapy. The theory of gene therapy is that doctors can treat a disorder by inserting a gene into the patient’s cells rather than using surgery or drugs. There are several approaches to gene therapy, including the following:

• When a mutated gene is functioning improperly, doctors use gene therapy to inactivate or knock out the malfunctioning gene.
• A healthy copy of the gene can replace a mutated gene that is currently causing disease.
• A new gene can be introduced into a body to assist in fighting disease.

Gene therapy is currently being tested for diseases for which there are no known cures. Gene therapy is promising on several fronts, including specific viral infections, certain types of cancers, and some inherited disorders.

What are the Goals of Gene Therapy and How Does It Work?

The goal of gene therapy is to introduce genetic material into a patient’s cells. This genetic material is introduced in an attempt to compensate for abnormal genes or to produce a beneficial protein. In some cases, a mutated gene can cause a necessary protein in the body to be flawed or to be missing altogether.

When a normal gene copy is introduced, the function of the necessary protein can potentially be restored. Genes are not introduced directly into a cell, because they do not function in this manner. Certain viruses may be used as the “vector” for introducing a gene into the body, as it then has direction and magnitude.

When genes are introduced via a virus vector, the virus is modified to avoid causing disease. Some types of viruses (like retroviruses) introduce the new gene into a chromosome in the human cell. Adenoviruses introduce DNA into the nucleus of the cell. The vector virus may be administered via IV into a specific tissue in the body.

Individual cells “take up” the gene. The patient’s cells may also be removed, exposed to the vector in a laboratory setting, then returned to the patient’s body. In this instance, when the gene therapy is successful, the new gene will create a properly functioning protein. The goal of gene therapy is to find better ways to deliver genes as well as better ways to target the genes to particular cells.

How Does Gene Therapy Help Patients?

Patients that have a certain disease could potentially be helped by replacing the mutated or defective gene with a healthy copy of the gene. Mutated genes that are not functioning properly, therefore causing disease in the body, can potentially be “fixed,” by turning the mutated gene off, or by introducing healthy genes that help the body fight the disease. Gene therapy can also work by exposing diseased cells to the immune system.

In some cases, the body fails to recognize cells that are a threat and can cause disease, so gene therapy “trains” the immune system to recognize these intruders. For patients who suffer from complex genetic disorders, gene therapy could truly be the light at the end of the tunnel. The gene therapies up for approval over the course of the next few years are largely focused on cancer treatments, while the others are meant to treat rare inherited genetic disorders. A gene therapy insurance denial can potentially stop patients from getting the treatments they need for their inherited genetic disorders.

Some of those genetic disorders—and their treatments—include:

Oncological disorders

Genetic testing for oncological disorders is often used to determine the likelihood of a person developing breast cancer, colon cancer, prostate cancer, pancreatic cancer, or ovarian cancer. Inherited cancers are those caused by a mutation in a gene that was present in the egg or sperm at the time of fertilization. These genetically-linked cancers make up a fairly small percentage of the cancers listed above, but can potentially identify these cancers in plenty of time to effectively treat them. Two relatively new treatments for these cancers are gene therapy and immunotherapy. Gene therapy uses genetic material to modify cells to help effect a cure. Gene therapies can decrease blood supply to a cancerous tumor, introduce genes into cancer cells that cause the death of those cancer cells or restore normal cellular phenotypes.

Retinal disorders

Retinitis pigmentosa is a common inherited disorder of the retina caused by a large number of genes with differing functions. Age-related macular degeneration—which has a genetic basis—is the most common cause of blindness in the United States. A gene therapy called Luxturna is a new breakthrough for those with inherited retinal dystrophy which is the result of two mutations of the gene RPE65. The cost of Luxturna is $850,000 for a one-time treatment, although the manufacturer is offering outcomes-based pricing for the drug in some cases.

Musculoskeletal disorders

Achondroplasia, Duchenne muscular dystrophy, and osteogenesis imperfecta are among the genetic disorders that affect the musculoskeletal system in children. These rare musculoskeletal genetic disorders are severely disabling, and can have significant impacts on families and children. The first drug to counter the genetic mutation that causes achondroplasia (the most common form of dwarfism) recently received accelerated FDA approval. A daily injection of BioMarin Pharmaceutical’s Voxzogo was approved for children who are five years old and older. In March 2024, the FDA approved Duvyzat, a nonsteroidal pill treatment for Duchenne muscular dystrophy for those 6 years and older. And in June 2024, the FDA expanded its approval for the gene therapy Elevidys, to nearly all patients with Duchenne muscular dystrophy.

Hematology/Blood Disorders

Genetically-linked blood disorders include acute myeloid leukemia, chronic myeloid leukemia, acute lymphoblastic leukemia, chronic lymphocytic leukemia, non-Hodgkin lymphoma, Hodgkin lymphoma, hemophilia, beta-thalassemia, and myeloma. A new therapy, betibeglogene autotemcel recently gained approval from the FDA as the first cell-based gene addition therapy for those with beta-thalassemia that require regular red blood cell transfusions. Beta-thalassemia can also be treated with a transplant of healthy stem cells from a matched sibling donor, however, this treatment option is not available to as many as three-quarters of those with the disorder. A new treatment for acute lymphoblastic leukemia is a drug called Blincyto (blinatumomab), which attaches to T-cells and cancer cells. The drug brings these cells close to one another to help the T-cells kill the cancer cells.

Cystic Fibrosis

Cystic fibrosis is a genetic disease caused when a baby inherits two copies of the defective cystic fibrosis gene (one copy from each parent). Both parents must have at least one copy of the defective gene to pass it along to their children, which means neither parent may actually have the disease if they each have only one copy. The newest treatment for cystic fibrosis is Trikafta, which addresses the root cause of the disease—mutations in the CFTR gene. New cystic fibrosis treatments are known as modulators because they help restore the function and shape of the CFTR protein. Trikafta has been found to significantly extend life expectancy for those with cystic fibrosis. At a list price of about $326,000 per year, it is quite expensive. There are currently two types of gene therapies that have the potential to treat cystic fibrosis, although the process of delivering the gene therapy technology to cells has its own set of challenges.

Fragile X Syndrome

This is a genetic disorder that affects an individual’s development, particularly the ability to learn, and behavioral issues. Fragile X can also affect sensitivity to light and noise, physical appearance, and communication skills. Fragile X Syndrome is the most common type of inherited developmental and intellectual disability. UB and Tetra Therapeutics are working toward a treatment for Fragile X Syndrome. The drug—BPN14770, achieved positive results in a phase 2 clinical study, significantly improving cognitive function in adult male patients with Fragile X. Gene therapy has shown promise in the treatment of Fragile X Syndrome. A functional FMR1 gene is delivered to cells to induce the production of FMRP proteins.

Sickle Cell Disease

Sickle cell disease is a group of inherited red blood cell disorders. In someone who has SCD, the hemoglobin is abnormal, causing the red blood cells to become hard and sticky. These sickle cells die early, resulting in a constant shortage of red blood cells, and a lack of oxygen to the body. When the sickle cells travel through smaller blood vessels, they can get stuck, reducing blood flow and causing such issues as strokes. Before December 2023, there were two primary treatments for sickle cell disease. The first is a bone marrow transplant, and the second is a new therapy known as LentiGlobin, which is a gene-based therapy where doctors harvest their patient’s own stem cells, adding them to a corrected gene that makes non-sickle hemoglobin. In December 2023, the FDA approved two gene therapies for this disease including Casgevy and Lyfgenia. Casgevy is the first FDA-approved prescription drug to use CRISPR gene editing technology. Casgevy might even be a cure for SCD.

Huntington’s Disease

As an inherited disorder that causes nerve cells in parts of the brain to break down and die, Huntington’s Disease primarily attacks the areas of the brain that control voluntary movement. One of the more recent drugs to treat cognitive changes associated with Huntington’s Disease is Sage-718, an NMDA receptor antagonist. Gene therapy drugs are also being tested for those with Huntington’s Disease which can potentially manage or slow the symptoms.

Tay-Sachs Disease

This is a rare genetic disorder that is passed from parents to child, caused by the absence of an enzyme that helps break down fatty substances called gangliosides that can build up to toxic levels in the brain and spinal cord, altering the function of nerve cells. While there is currently no treatment for Tay-Sachs, gene therapy is being researched to treat the disease by delivering working HEXA and HEXB genes into the cells via a viral vector. Two babies have recently received the first-ever gene therapy for Tay-Sachs after fourteen years of development of the therapy.

Early-onset Alzheimer’s Disease

Researchers have found several genes that increase the risk of Alzheimer’s disease. APOE-e4 is the first risk gene identified, remaining the gene with the strongest impact on risk. Between 40 and 65 percent of those diagnosed with Alzheimer’s have this gene, inherited from a parent. Those who inherit one copy of APOE-e4 from one parent have an increased risk of developing Alzheimer’s, while those with two copies (one from each parent) have an even higher risk. Gene therapy drugs are currently being tested as a treatment for inherited Alzheimer’s disease.

Hemochromatosis

Hereditary hemochromatosis is caused by variants in the HFE gene. When an individual inherits two of these variants (one from each parent), they will have hereditary hemochromatosis and are at risk of developing high iron levels that can cause extreme tiredness and weakness. Deferoxamine (Desferal) is a drug that binds to iron, allowing it to be removed from the body in those with hemochromatosis. One new gene therapy for hemochromatosis is the inhibition of DMT1 synthesis by antisense-generating genes. This gene therapy has been shown to markedly inhibit iron uptake by intestinal epithelial cells.

Are There Gene Therapy Drugs Currently Approved by the FDA?

Some forms of gene therapy have been approved by the FDA while others are available primarily in a research setting. (An estimated 1.09 million patients will be treated with a gene therapy by the end of 2034.)  There are hundreds of clinical trials currently underway that are testing gene therapy for use as a cancer treatment, genetic conditions, and even HIV/AIDS. If you are interested in taking part in a clinical trial that uses gene therapy, you can search online at ClinicalTrials.gov. The FDA website has an entire list of gene therapy drugs that have been approved by the FDA. The approved gene therapy drugs include:

• ABECMA (idecabtagene vicleucel) – Celgene Corporation, a Bristol-Myers Squibb Company
• ADSTILADRIN (nadofaragene firadenovec-vncg) – Ferring Pharmaceuticals A/S
• ALLOCORD (HPC, Cord Blood) – SSM Cardinal Glennon Children’s Medical Center
• AMTAGVI (lifileucel) – Iovance Biotherapeutics, Inc.
• BEQVEZ (fidanacogene elaparvovec-dzkt) – Pfizer, Inc.
• BREYANZI – Juno Therapeutics, Inc., a Bristol-Myers Squibb Company
• CARVYKTI (ciltacabtagene autoleucel) – Janssen Biotech, Inc.
• CASGEVY (exagamglogene autotemcel [exa-cel]) – Vertex Pharmaceuticals Incorporated
• CLEVECORD (HPC Cord Blood) – Cleveland Cord Blood Center
• Ducord, HPC Cord Blood – Duke University School of Medicine
• ELEVIDYS (delandistrogene moxeparvovec-rokl) – Sarapeta Therapeutics, Inc.
• GINTUIT (Allogeneic Cultured Keratinocytes and Fibroblasts in Bovine Collagen) – Organogenesis Incorporated
• HEMACORD (HPC, cord blood) – New York Blood Center
• HEMGENIX – CSL Behring LLC
• HPC, Cord Blood – Clinimmune Labs, University of Colorado Cord Blood Bank
• HPC, Cord Blood – MD Anderson Cord Blood Bank – MD Anderson Cord Blood Bank
• HPC, Cord Blood – LifeSouth – LifeSouth Community Blood Centers, Inc.
• HPC, Cord Blood – Bloodworks – Bloodworks
• IMLYGIC (talimogene laherparepvec) – BioVex, Inc., a subsidiary of Amgen Inc.
• KYMRIAH (tisagenlecleucel) – Novartis Pharmaceuticals Corporation
• LANTIDRA (donislecel) – CellTrans Inc.
• LAVIV (Azficel-T) – Fibrocall Technologies
• LENMELDY (atidarsagene autotemcel) – Orchard Therapeutics (Europe) Limited
• LUXTURNA – Spark Therapeutics, Inc.
• LYFGENIA (lovotibeglogene autotemcel [lovo-cel]) – bluebird bio, Inc.
• MACI (Autologous Cultured Chondrocytes on a Porcine Collagen Membrane) – Vericel Corp.
• PROVENGE (sipuleucel-T) – Dendreon Corp.
• RETHYMIC – Enzyvant Therapeutics GmbH
• SKYSONA (elivaldogene autotemcel) – bluebird bio, Inc.
• STRATAGRAFT – Stratatech Corporation
• TECARTUS (brexucabtagene autoleucel) – Kite Pharma, Inc.
• YESCARTA (axicabtagene ciloleucel) – Kite Pharma, Incorporated
• ZYNTEGLO (betibeglogene autotemcel) – blubird bio, Inc.
• ZOLGENSMA (onasemnogene abeparvovec-xioi) – AveXis, Inc.

Approved Non-Cancer Gene Therapies

Gene therapy, which involves replacing a defective gene in the body with a healthy copy of that gene, is primarily used to treat different types of cancers. Chemotherapy, the traditional cancer treatment, lacks the specificity that gene therapy offers. In the past year, there have been multiple developments in gene therapy for medical issues other than cancer. These include:

• Zynteglo (Beta thalassemia gene therapy) is used for those who have mutations in the beta-globin gene, a key hemoglobin component. Adults and children who require regular transfusions of red blood cells can benefit from Zynteglo, which gained FDA approval in August 2022. Zynteglo delivers a functioning copy of the beta-globin gene into patients’ blood stem cells.
• Luxturna is a gene therapy that treats those with an inherited retinal dystrophy due to two mutations of the RPE65 gene.
• Skysona is a one-time gene therapy used to treat boys with CALD—early, active cerebral adrenoleukodystrophy—which is a genetic disease that is the result of ABCD1genetic mutations. ABCD1 is the gene that leads to the buildup of very long-chain fatty acids in the brain. CALD affects the brain of boys during childhood, occurring in about one in 21,000 boys between the ages of 4 and 10. While females may also have the ABCD1 mutation, they are unlikely to develop CALD.
• Zolgensma is a one-time gene therapy that halts the progression of SMA (spinal muscular atrophy). SMA occurs in children who are born with a missing or defective SMN1 gene. Zolgensma delivers a new, working SMN1 gene to the motor neuron cells, which allows the muscles to work as they should.
• In June 2023, the FDA approved Elevidys, the first gene therapy drug for children aged 4-5 with Duchenne muscular dystrophy. Children who can receive this treatment have a mutation of the DMD gene and have no pre-existing medical reason that prevents treatment with Elevidys.

Through the end of the first quarter of 2023, there were more than 100 different approved gene, cell, and RNA therapies across the globe, with more than 3,700 gene therapies currently in clinical and preclinical development. According to the FDA, there are continuous discoveries in gene therapies regarding how gene therapy works and expanding the use of gene therapy beyond cancer treatment. It is expected that a wide range of challenging diseases may be treated with gene therapy in the future, particularly serious, rare diseases. Gene therapy will soon become a mainstay in the treatment of these diseases, potentially curing many of the most devastating diseases.

The FDA intends to establish a suite of disease-specific guidance documents regarding the development of new gene therapy products. New medical research has shown promising results regarding the potential of gene therapy to cure genetic conditions like sickle cell disease, so this is likely to be on the horizon in the near future. In fact, six patients with sickle cell disease have undergone gene therapy as a part of a major clinical trial at Boston’s Children’s Hospital and the results of these trials should be available soon.

Why Would Insurance Deny Gene Therapy?

Despite the fact that amazing breakthroughs have recently occurred in the gene therapy field, these treatments can be expensive, causing many insurance companies to deny the treatment. That being said, insurance companies are slowly beginning to change their policies, thus allowing more people to receive these potentially life-saving treatments. Like many new treatments, gene therapy was once considered largely experimental, so it is taking time and information for insurance companies to reverse their policies regarding gene therapy treatments.

Currently, some insurance companies will approve gene therapy, but usually only for illnesses with no other known cures or treatments. One such disease is spinal muscular atrophy, a genetic disorder characterized by atrophy of skeletal muscles. When spinal muscular atrophy occurs, the nerve cells that control movement are lost, worsening over time, and significantly shortening a person’s lifespan.

Gene therapy may also be approved—depending on your insurance company—to treat a rare genetic vision disorder in children and adults. Luxturna was approved by the FDA in 2017 for treating this vision disorder by targeting vision loss caused by gene mutations. Zolgensma is another gene therapy treatment drug that treats children under the age of two with genetically linked spinal muscular atrophy (SMA). Zolgensma targets and repairs missing or non-functioning genes responsible for SMA. In the case of Zolgensma, the treatment must be administered as quickly as possible, before damage from SMA accumulates and is irreversible.

What Happens Following a Gene Therapy Drug Insurance Denial?

While it is not particularly unusual for insurers to deny certain claims, or refuse to cover a test, procedure, or service that your doctor has ordered, these decisions can definitely be appealed. Medicare claim denials can also be appealed. Once a claim is denied, you might ask for more information from a case manager or customer service rep before making a formal appeal. This information will also help your gene therapy drug insurance denial attorney fight the decision.

Your insurance company is required by law to tell you why your doctor’s request for a gene therapy drug insurance was denied and to tell you how long you have to file an internal appeal. In some cases—although gene therapy is unlikely to be one of these cases—you can re-submit your claim with a copy of your denial letter and information from your doctor that supports the treatment to have it coded differently.

If an internal appeal is unsuccessful, your next step is a formal appeal. It can be difficult to go through all the proper steps of a formal appeal if you are not familiar with the process. Having an experienced insurance denial attorney like Scott Glovsky on your side can truly make all the difference in the final outcome. Make sure you keep every piece of correspondence from your insurance company, as well as a log of dates, names, and conversations you have had regarding the denial.   

How the Law Offices of Scott Glovsky Can Help with a Gene Therapy Drug Insurance Denial

If you or a loved one in California have had a gene therapy drug insurance denial, attorney Scott Glovsky understands the urgency of the situation and that this gene therapy could be your only hope for recovery. Scott helps clients with health insurance denials based on experimental or investigational treatments and will work to examine the reasons your claim was denied, investigate relevant case law, file the appropriate appeals, and go to court on your behalf. With offices in Claremont and Pasadena, we can help residents across the state of California fight insurance denials. Contact the Law Offices of Scott Glovsky today to get the help you need and deserve for a brighter future.