Vertex Presents Positive Long-Term Data On CASGEVY™ (exagamglogene autotemcel) at the American Society of Hematology (ASH) Annual Meeting and Exposition and Provides Program Update
VRTX 12.08.2024
Drug:exagamglogene-autotemcel CASGEVY
Diseases:Sickle Cell Disease
Diseases:Transfusion-Dependent Beta Thalassemia
Date of Upcoming Event:2024-12-08
Name of Upcoming Event:American Society of Hematology (ASH) Annual Meeting and Exposition
About Gravity Analytica
- Data from long-term follow-up of patients in clinical trials further demonstrate durability of the transformative benefits of CASGEVYTM -
-Safety profile consistent with busulfan conditioning andautologous hematopoietic stem cell transplant -
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The results, presented at the
“These comprehensive data provide additional evidence of the benefits of eradicating transfusion requirements for people with transfusion-dependent beta thalassemia and vaso-occlusive crises for those with sickle cell disease,” said
“CASGEVY is changing the outlook for people living with sickle cell disease and beta thalassemia, with these data reinforcing the immense clinical value a durable one-time therapy can provide to patients,” said
New long-term follow-up data presented from the CASGEVY trials
- In SCD, 39/42 (93%) evaluable patients (those with at least 16 months of follow-up) were free from vaso-occlusive crises (VOCs) for at least 12 consecutive months (VF12) in CLIMB-121 and CLIMB-131 combined. The mean duration of VOC-free was 30.9 months, with a maximum of 59.6 months.
- The three evaluable patients who have not achieved VF12 have derived meaningful clinical benefit including by reducing their rate of hospitalization for VOCs by 91%, 71% and 100%.
- In TDT, 53/54 (98%) evaluable patients (those with at least 16 months of follow-up) achieved transfusion-independence for at least 12 consecutive months with a weighted average hemoglobin of at least 9 g/dL (TI12) in CLIMB-111 and CLIMB-131 combined. The mean duration of transfusion independence was 34.5 months, with a maximum of 64.1 months.
- The one evaluable patient who has not yet achieved TI12 has been transfusion free for 8.2 months.
- Both SCD and TDT patients reported sustained and clinically meaningful improvements in their quality of life, including physical, emotional, social/family and functional well-being, and overall health status.
- The safety profile of CASGEVY continues to be generally consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant.
- Patients continue to demonstrate stable levels of fetal hemoglobin (HbF) and allelic editing across all ages and genotypes in the trials.
- Oral presentation, Abstract #512, entitled “Durable Clinical Benefits with Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia”
- Poster presentation, Abstract #4954, entitled “Durable Clinical Benefits with Exagamglogene Autotemcel for Severe Sickle Cell Disease”
- Poster presentation, Abstract #1098, entitled “Estimated Prevalence of β-Thalassemia in
the United States in 2023” - Publication only, Abstract #7454, entitled “Health-Related Quality-of-Life Improvements after Exagamglogene Autotemcel in Patients with Transfusion-Dependent Beta Thalassemia”
- Publication only, Abstract #7453, entitled “Health-Related Quality-of-Life Improvements after Exagamglogene Autotemcel in Patients with Severe Sickle Cell Disease”
- Publication only, Abstract #7660, entitled “Adherence, Clinical and Economic Outcomes in Patients with Sickle Cell Disease with Recurrent Vaso-Occlusive Crises Treated with L-Glutamine, Voxelotor, or
Crizanlizumab Covered By Medicaid and Commercial Insurance in the United States” - Publication only, Abstract #7661, entitled “Clinical Complications and Healthcare Resource Utilization in Medicaid and Commercially Insured Patients with Sickle Cell Disease Receiving Frequent Red Blood Cell Transfusions”
Progress in bringing CASGEVY to patients around the world
CASGEVY is approved for both SCD and TDT in the
About Sickle Cell Disease (SCD)
SCD is a debilitating, progressive and life-shortening disease. SCD patients report health-related quality of life scores well below the general population, and the lifetime health care costs in the
About Transfusion-Dependent Beta Thalassemia (TDT)
TDT is a serious, life-threatening genetic disease. TDT patients report health-related quality of life scores below the general population and the lifetime health care costs in the
About CASGEVYTM (exagamglogene autotemcel [exa-cel])
CASGEVYTM is a non-viral,ex vivoCRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of theBCL11Agene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT.
CASGEVY is approved for eligible SCD and TDT patients 12 years and older by multiple regulatory bodies around the world.
About the CLIMB Trials
The ongoing Phase 1/2/3 open-label trials, CLIMB-111 and CLIMB-121, are designed to assess the safety and efficacy of a single dose of CASGEVY in patients ages 12 to 35 years with TDT or with SCD and recurrent VOCs. The trials are closed for enrollment. Patients will be followed for approximately two years after CASGEVY infusion in these trials. Each patient will be asked to participate in the ongoing long-term, open-label trial, CLIMB-131. CLIMB-131 is designed to evaluate the long-term safety and efficacy of CASGEVY in patients who received CASGEVY, including those in other CLIMB trials. The trial is designed to follow patients for up to 15 years after CASGEVY infusion.
WHAT IS CASGEVY?
CASGEVY is a one-time therapy used to treat people aged 12 years and older with:
• sickle cell disease (SCD) who have frequent vaso-occlusive crises or VOCs
• beta thalassemia (β-thalassemia) who need regular blood transfusions
CASGEVY is made specifically for each patient, using the patient’s own edited blood stem cells, and increases the production of a special type of hemoglobin called hemoglobin F (fetal hemoglobin or HbF). Having more HbF increases overall hemoglobin levels and has been shown to improve the production and function of red blood cells. This can eliminate VOCs in people with sickle cell disease and eliminate the need for regular blood transfusions in people with beta thalassemia.
IMPORTANT SAFETY INFORMATION
What is the most important information I should know about CASGEVY?
After treatment with CASGEVY, you will have fewer blood cells for a while until CASGEVY takes hold (engrafts) into your bone marrow. This includes low levels of platelets (cells that usually help the blood to clot) and white blood cells (cells that usually fight infections). Your doctor will monitor this and give you treatment as required. The doctor will tell you when blood cell levels return to safe levels.
- Tell your healthcare provider right awayif you experience any of the following, which could be signs of low levels of platelet cells:
- severe headache
- abnormal bruising
- prolonged bleeding
- bleeding without injury such as nosebleeds; bleeding from gums; blood in your urine, stool, or vomit; or coughing up blood
- Tell your healthcare provider right awayif you experience any of the following, which could be signs of low levels of white blood cells:
- fever
- chills
- infections
You may experience side effects associated with other medicines administered as part of the treatment regimen for CASGEVY. Talk to your physician regarding those possible side effects. Your healthcare provider may give you other medicines to treat your side effects.
How will I receive CASGEVY?
Your healthcare provider will give you other medicines, including a conditioning medicine, as part of your treatment with CASGEVY. It’s important to talk to your healthcare provider about the risks and benefits of all medicines involved in your treatment.
After receiving the conditioning medicine, it may not be possible for you to become pregnant or father a child. You should discuss options for fertility preservation with your healthcare provider before treatment.
STEP 1:Before CASGEVY treatment, a doctor will give you mobilization medicine(s). This medicine moves blood stem cells from your bone marrow into the blood stream. The blood stem cells are then collected in a machine that separates the different blood cells (this is called apheresis). This entire process may happen more than once. Each time, it can take up to one week.
During this step rescue cells are also collected and stored at the hospital. These are your existing blood stem cells and are kept untreated just in case there is a problem in the treatment process. If CASGEVY cannot be given after the conditioningmedicine, or if the modified blood stem cells do not take hold (engraft) in the body, these rescue cells will be given back to you. If you are given rescue cells, you will not have any treatment benefit from CASGEVY.
STEP 2:After they are collected, your blood stem cells will be sent to the manufacturing site where they are used to make CASGEVY. It may take up to 6 months from the time your cells are collected to manufacture and test CASGEVY before it is sent back to your healthcare provider.
STEP 3:Shortly before your stem cell transplant, your healthcare provider will give you a conditioning medicine for a few days in hospital. This will prepare you for treatment by clearing cells from the bone marrow, so they can be replaced with the modified cells in CASGEVY. After you are given this medicine, your blood cell levels will fall to very low levels. You will stay in the hospital for this step and remain in the hospital until after the infusion with CASGEVY.
STEP 4:One or more vials of CASGEVY will be given into a vein (intravenous infusion) over a short period of time.
After the CASGEVY infusion, you will stay in hospital so that your healthcare provider can closely monitor your recovery. This can take 4-6 weeks, but times can vary. Your healthcare provider will decide when you can go home.
What should I avoid after receiving CASGEVY?
- Do not donate blood, organs, tissues, or cells at any time in the future
What are the possible or reasonably likely side effects of CASGEVY?
The most common side effects of CASGEVY include:
- Low levels of platelet cells, which may reduce the ability of blood to clot and may cause bleeding
- Low levels of white blood cells, which may make you more susceptible to infection
Your healthcare provider will test your blood to check for low levels of blood cells (including platelets and white blood cells). Tell your healthcare provider right away if you get any of the following symptoms:
- fever
- chills
- infections
- severe headache
- abnormal bruising
- prolonged bleeding
- bleeding without injury such as nosebleeds; bleeding from gums; blood in your urine, stool, or vomit; or coughing up blood
These are not all the possible side effects of CASGEVY. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
General information about the safe and effective use of CASGEVY
Talk to your healthcare provider about any health concerns.
Please see fullPrescribing InformationincludingPatient Informationfor CASGEVY.
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