In December 2023, the U.S. Food and Drug Administration approved two treatments,
Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment
of sickle cell disease (SCD) in patients 12 years and older.
Casgevy, a cell-based gene therapy, is approved for the treatment of sickle cell disease
in patients 12 years of age and older with recurrent vaso-occlusive crises.
Lyfgenia is a cell-based gene therapy.
Lyfgenia uses a lentiviral vector (gene delivery
vehicle) for genetic modification and is approved for the treatment of patients 12 years
of age and older with sickle cell disease and a history of vaso-occlusive events.
Both products are made from the patients’ own blood stem cells, which are modified,
and are given back as a one-time, single-dose infusion as part of a hematopoietic
(blood) stem cell transplant. Prior to treatment, a patients’ own stem cells are collected,
and then the patient must undergo a conditioning regimen with chemotherapy; a
process that removes cells from the bone marrow so they can be replaced with the
modified cells in Casgevy and Lyfgenia.
When considering therapy options for sickle cell disease, patients should ask their
healthcare providers the following important questions:
• What are all the available treatment options for my specific case of sickle cell
disease?
• How do the different treatments compare in terms of effectiveness and potential
side effects?
• Am I a candidate for gene therapy?
• What are the potential benefits and risks of the two approved gene therapies?
• How do the two FDA-approved gene therapies (CASGEVY and LYFGENIA)
differ?
• What is the success rate of these gene therapies in clinical trials?
• What does the treatment process involve, and how long does it take?
• What is the expected recovery time, and how will it impact my daily life?
• Will I need to be hospitalized, and for how long?
• What are the potential long-term effects of the treatment?
• How will the treatment affect my fertility and reproductive options?
• Will I need ongoing monitoring or follow-up treatments
• Is this treatment covered by my insurance?
• What out-of-pocket costs should I expect?
• Are there any clinical trials I might be eligible for?
• How will this treatment potentially improve my quality of life?
• Will I still need to take other medications after the treatment?
• What is your experience with this treatment?
• Can you refer me to a specialist or center that has expertise in these new
therapies?
• What support services are available during and after treatment?
It is important for patients and their caregivers to ask their care providers these
questions to gain a comprehensive understanding of their treatment options and make
informed decisions about their care, especially with the approval and availability of
potentially curative gene therapies.