What is Lyfgenia?

Lyfgenia is a recently FDA-approved cell-based gene therapy from Bluebird Bio for adult and pediatric patients with sickle cell disease. Lyfgenia uses a lentiviral vector for genetic modification and is a one-time treatment approved for people 12 years old or older with recurrent vaso-occlusive events.

Children’s Health is one of a few designated Qualified Treatment Centers (QTCs) in the country able to provide Lyfgenia.

What are the potential risks or side effects associated with Lyfgenia?

The most common side effects for Lyfgenia are stomatitis (mouth sores of the lips, mouth, and throat), low levels of platelets, white blood cells, and red blood cells, and fever and low white blood cell count).

Hematologic malignancy (blood cancer) has also occurred in some patients treated with Lyfgenia. After a thorough investigation, the blood cancer was not found to be caused by the gene therapy itself. A black box warning is included in the label for Lyfgenia with information regarding this risk.

How can a child receive this treatment?

Lyfgenia is only available at Qualified Treatment Centers (QTCs). Children’s Health is one of a few designated centers in the country able to provide this gene therapy. This treatment is recommended for children aged 12 years old or older. To receive this gene therapy, your child must be seen in our CCBD clinic to determine if Lyfgenia is the appropriate treatment.

If your child is eligible for Lyfgenia, our CCBD Clinic and Centralized Authorization Department team will work with your insurance company to obtain prior authorization.

As of today, the following insurers have established coverage guidelines for Lyfgenia. We are awaiting guidelines from all other insurance carriers and we are hopeful additional guidelines will be released soon.

If your insurer has not issued guidelines, here are two things you can do:

1. We encourage you to contact your insurance carrier and advocate for your child to receive Lyfgenia

2. If your insurance plan is through an employer, we also suggest you contact your employer’s HR personnel and ask them to also advocate for your child with contacts they may have with your insurance provider.

   • If you are a Texas Medicaid recipient, we recommend that you call and advocate for coverage.

We know this can be a challenging waiting process as insurance carrier guidelines are created. Children’s Health will continue to stay updated on new information so that we can support our patient families in their steps toward receiving Lyfgenia.

If you have additional questions, please contact a Children’s Health Hematologist in the CCBD Clinic at 214-456-2382.