February 1, 2024

A New Generation of Treatment for Sickle Cell Disease

On December 8, 2023, the US Food and Drug Administration (FDA) approved two groundbreaking treatments.

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For individuals with sickle cell disease (SCD), managing the disease involves treating pain episodes and preventing further complications such as infection, stroke, and organ damage. Many practitioners rely on disease-modifying agents and blood transfusions as needed to help their patients to do this.

According to the Center for Disease Control and Prevention, approximately 10,000 Americans suffer from SCD with the only known, potential cure is a bone marrow transplant. However, finding a match can be difficult, and it is not guaranteed to work for everyone. New developments with gene therapies that involve either restoring a faulty or missing gene or adding a new gene to improve the way the cell works has brought new hope for a cure. 

New Therapy Update 

On December 8, 2023, the US Food and Drug Administration (FDA) approved two groundbreaking treatments that alter genes to reduce the mutation in red blood cells, representing the first cell-based gene therapies for the treatment of SCD. 

Both of these new treatments are made from the patients’ own blood stem cells, which are collected, modified, and given back as a one-time, single-dose infusion as part of a hematopoietic (blood) stem cell transplant.


Developed by Vertex Pharmaceutical Inc., Casgevy uses gene editing technology to reduce BCL11A expression, the gene that is responsible for inhibiting gamma-globin and the production of functional fetal hemoglobin. The FDA approved treatment is for patients 12 years of age and older who suffer from recurrent vaso-occlusive crises (VOCs). 

Casgevu is the first FDA-approved therapy that uses the innovative genome editing technology CRISPR/Cas9.

While the estimated cost for the one-time infusion of Casgevy is $2,200,000, it is intended to address the underlying cause of SCD to eliminate VOCs and the increased medical costs associated with it.  

In a clinical trial, 44 patients ages 12 to 35 received the treatment. Results show that 93.5% of evaluable patients experienced freedom from severe VOC episodes for at least 12 consecutive months during the 24-month follow-up period. The most common side effects include mucositis, febrile neutropenia, decreased appetite, neutropenia, thrombocytopenia, leukopenia, anemia, and lymphopenia. 

Patients who receive Casgevy will be required to do routine follow-up lab work, monitoring, infection prophylaxis, and blood cell support. They will also be followed in a long-term study to evaluate the product’s safety and effectiveness.


Produced by bluebird bio, Lyfgenia is also approved for the treatment of patients 12 years of age and older who have SCD and a history of VOCs. It is a cell-based gene therapy that uses the lentiviral vector method for genetic modification by introducing functional copies of a modified  βA-globin gene into patients’ hematopoietic stem cells. 

Currently, the estimated cost for the one-time infusion of Lyfgenia is $3,100,000. The intended goal of the treatment is to address the underlying cause of SCD, eliminate VOCS, and as a result, eliminate the increased complications and medical costs associated with the disease. 

In a clinical trial, 32 patients between the ages of 12 and 50 received the treatment. Of those patients, 88% experienced a complete resolution of vaso-occlusive events between six and 18 months after infusion, which is the intended outcome of Lyfgenia. The most common side effects reported were stomatitis, thrombocytopenia, neutropenia, febrile neutropenia, anemia and leukopenia. There is also a risk that patients with the α-thalassemia trait may experience anemia that may require chronic red blood cell transfusions.

Patients who receive Lyfgenia must participate in routine follow-up lab work, monitoring, infection prophylaxis, and blood cell support and will be followed in a long-term study to evaluate the treatment’s safety and effectiveness. 

What is the Clinical Impact?

These two therapies represent the first-ever gene therapist to potentially cure a hereditary condition. For patients, this has the potential to be a game changer as these new therapies aim to cure the disease as opposed to controlling symptoms. Providers are also able to offer hope to their patients with SCD that they previously could not. 

What is the Financial Impact for Self-Funded Health Plans?

While the costs for Casgevy and Lyfgenia are estimated to be 2.2 million and 3.1 million respectively, this does NOT include pre-treatment and post-treatment hospitalization of 4-6 weeks or any complications that may arise. Although the risk for these two treatments (and their associated claims) remains extremely low for a self-funded group plans, it’s critical to have a thorough risk mitigation program in place. Especially as more gene therapies emerge for both rare and more prevalent diseases.

To navigate SCD and other gene therapy risks, self-funded group plans require a healthcare partner with deep experience implementing appropriate cost savings techniques, patient support protocols, and reviewing and auditing current policies based on emerging trends. 

Healthgram’s Approach to Gene Therapies & Emerging Treatments

At Healthgram, we take a multi-pronged approach to managing these new gene therapies and other emerging treatments. This includes in-house clinical oversight, unique plan language, alternative funding analysis, and seamless reinsurance integration. Our clinical staff and claim experts continue to monitor the market and future risk levels of emerging treatments for clients.  

Vice President of Pharmacy Benefits Bryan Klazinga explained, “We will continue to advise our broker partners and clients about strategies to minimize gene therapy risk exposure. This includes the appropriateness of risk sharing and alternative funding programs.  If a patient of a customer is prescribed this treatment, we would follow a detailed protocol that we have created for all high-dollar treatments like gene therapies. It’s critical for all parties involved (the patient, customer, broker, and reinsurance carrier) that we provide the right treatment for the right patient at the right cost.” 

To learn more about how Healthgram can improve your company’s self-funded approach, reach out to a member of our team.