Getting your patients started with CASGEVY®

Getting your patients started with CASGEVY®

CASGEVY is indicated for the treatment of patients aged 12 years and older with transfusion-dependent β-thalassemia (TDT)1

CASGEVY is indicated for the treatment of patients aged 12 years and older with transfusion-dependent β-thalassemia (TDT)1

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Approved for all genotypes

CASGEVY was studied in patients with β0/β0-like or non-β0/β0-like.1

CASGEVY was studied in patients with β0/β0-like or non-β0/β0-like.1

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Autologous

No donor is needed because CASGEVY uses a patient’s own cells.1

Identifying potential candidates with TDT for CASGEVY:

Identifying potential candidates with TDT for CASGEVY:

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Age Consideration:

Is my patient at least 12 years of age?

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Treatment Challenges:

How often is my patient experiencing RBC transfusions per year?

How often is my patient experiencing RBC transfusions per year?

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Treatment Preferences:

Has my patient shown interest in exploring new treatments or gene-therapy options?

Has my patient shown interest in exploring new treatments or gene therapy options?

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Support Assessment:

How equipped is my patient with a supportive network to navigate their treatment journey effectively?

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Physical Condition:

Is my patient able to tolerate a stem cell transplant?

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Life Stage Consideration:

Is my patient at a suitable stage in their life to embark on the treatment journey with CASGEVY?

Is my patient at a suitable stage in their life to embark on the treatment journey with CASGEVY?

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Fertility Considerations*:

Does my patient have plans to start a family in the future?

Does my patient have plans to start a family in the future?

*Infertility has been observed with myeloablative conditioning therefore, advise patients of fertility preservation options before treatment, if appropriate.1

By considering these factors, healthcare providers can collaborate with their patients to tailor treatment approaches effectively.

By considering these factors, healthcare providers can collaborate with their patients to tailor treatment approaches effectively.

Potential candidates for CASGEVY

These are hypothetical profiles and medical providers should make their own independent decisions regarding the treatment decisions for their patients.

Edward profile
    Edward (Adolescent)

    Meet Edward, a sociable 12-year-old whose participation in his school’s coding club is sometimes limited by his TDT treatment schedule

    Meet Edward, a sociable 12-year-old whose participation in his school’s coding club is sometimes limited by his TDT treatment schedule

    • Diagnosed at 1 year of age (non-β0/β0-like genotype); inconsistent adherence to iron chelation therapy
    • Prior to transfusions, often feels very tired; frustrated when he has to miss class and club meetings with friends to receive transfusions
    • Involved parents have expressed concern about symptom progression. They are curious about gene therapy and its effects on Edward’s future fertility
    • Diagnosed at 1 year of age (non-β0/β0-like genotype); inconsistent adherence to iron chelation therapy
    • Prior to transfusions, often feels very tired; frustrated when he has to miss class and club meetings with friends to receive transfusions
    • Involved parents have expressed concern about symptom progression. They are curious about gene therapy and its effects on Edward’s future fertility
    Doctor and patient icon Doctor and patient icon Doctor and patient icon

    Treatment History

    Treatment History

    • Current treatments: Iron chelation (deferasirox 1x daily), blood transfusions every 4 weeks (2 units)
    • Transplantation status: No donor (matched or unrelated) available
    • Current treatments: Iron chelation (deferasirox 1x daily), blood transfusions every 4 weeks (2 units)
    • Transplantation status: No donor (matched or unrelated) available
    Tranfusion icon Tranfusion icon Tranfusion icon

    Experience with TDT

    Experience with TDT

    • RBC transfusions per year in the last 2 years: 13 (26 units/year)
    • Comorbidities: None
    • End-organ damage: None
    • RBC transfusions per year in the last 2 years: 13 (26 units/
      year)
    • Comorbidities: None
    • End-organ damage: None

    Edward’s last 24 months with TDT

    (Number of transfusions comparable in both years)

    Edward’s last 24 months with TDT

    (Number of transfusions comparable in both years)

      RBC transfusion
    Edward Timeline
      RBC transfusion

    Edward’s selected lab values

    (At last visit; values have fluctuated over the last year)

    Edward’s selected lab values

    (At last visit; values have fluctuated over the last year)

    Selected lab values

    • Total hemoglobin (g/dL): 9.1 | Reference range: M: 14-18
    • AST (U/L): 22 | Reference range: 10-40
    • AST (U/L): 15 | Reference range: 10-40
    • Creatinine (mg/dL): 0.48 | Reference range: M: 0.70-1.30
    • Ferritin (ng/mL): 782 | Reference range: M: 24-336

    Selected lab values

    • Total hemoglobin (g/dL): 9.1 | Reference range: M: 14-18
    • AST (U/L): 22 | Reference range: 10-40
    • AST (U/L): 15 | Reference range: 10-40
    • Creatinine (mg/dL): 0.48 | Reference range: M: 0.70-1.30
    • Ferritin (ng/mL): 782 | Reference range: M: 24-336

    Radiology

    • Liver iron concentration (mg/g): 2.6 | Reference range: ≤1.8
    • Cardiac T2* (msec): 40.1 | Reference range: >20

    Radiology

    • Liver iron concentration (mg/g): 2.6 | Reference range: ≤1.8
    • Cardiac T2* (msec): 40.1 | Reference range: >20
    Patient Brochure icon Patient Brochure icon Patient Brochure icon

    TDT Patient Brochure

    TDT Patient
    Brochure

    Consider walking a patient like Edward and their family through the CASGEVY TDT Patient Brochure so they can begin to educate themselves on the clinical study results, safety, and side effects of CASGEVY and decide if CASGEVY may be right for them.

    Consider walking a patient like Edward and their family through the CASGEVY TDT Patient Brochure so they can begin to educate themselves on the clinical study results, safety, and side effects of CASGEVY and decide if CASGEVY may be right for them.

    Amina profile
      Amina (Adult)

      Meet Amina, an ambitious 19-year-old chemistry major on the honors list with a regular transfusion schedule to manage her TDT

      • Diagnosed at birth (β0/β0-like genotype)
      • Diligent about adherence
      • Increasingly worried about missing classes for transfusions and the impact of her current treatment schedule on her demanding studies

      Meet Amina, an ambitious 19-year-old chemistry major on the honors list with a regular transfusion schedule to manage her TDT

      • Diagnosed at birth (β0/β0-like genotype)
      • Diligent about adherence
      • Increasingly worried about missing classes for transfusions and the impact of her current treatment schedule on her demanding studies
      Doctor and patient icon Doctor and patient icon Doctor and patient icon

      Treatment History

      Treatment History

      • Current treatments: Iron chelation (deferiprone 2x daily), blood transfusions every 3 weeks (2 units)
      • Transplantation status: No donor (matched or unrelated) available
      • Current treatments: Iron chelation (deferiprone 2x daily), blood transfusions every 3 weeks (2 units)
      • Transplantation status: No donor (matched or unrelated) available
      Tranfusion icon Tranfusion icon Tranfusion icon

      Experience with TDT

      Experience with TDT

      • RBC transfusions per year in the last 2 years: 17 (34 units/year)
      • Comorbidities: Splenomegaly
      • RBC transfusions per year in the last 2 years: 17 (34 units/
        year)
      • Comorbidities: Splenomegaly

      Amina’s last 24 months with TDT

      (Number of transfusions comparable in both years)

      Amina’s last 24 months with TDT

      (Number of transfusions comparable in both years)

        RBC transfusion
      Amina timeline
        RBC transfusion

      Amina’s selected lab values

      (At last visit; values have fluctuated over the last year)

      Selected lab values

      • Total hemoglobin (g/dL): 10.1 | Reference range: F: 12-16
      • AST (U/L): 24 | Reference range: 10-40
      • ALT (U/L): 14 | Reference range: 10-40
      • Creatinine (mg/dL): 0.56 | Reference range: F: 0.50-1.10
      • Ferritin (ng/mL): 362 | Reference range: F: 24-307

      Selected lab values

      • Total hemoglobin (g/dL): 10.1 | Reference range: F: 12-16
      • AST (U/L): 24 | Reference range: 10-40
      • ALT (U/L): 14 | Reference range: 10-40
      • Creatinine (mg/dL): 0.56 | Reference range: F: 0.50-1.10
      • Ferritin (ng/mL): 362 | Reference range: F: 24-307

      Radiology

      • Liver iron concentration (mg/g): 2.2 | Reference range: ≤1.8
      • Cardiac T2* (msec): 31.9 | Reference range: >20

      Radiology

      • Liver iron concentration (mg/g): 2.2 | Reference range: ≤1.8
      • Cardiac T2* (msec): 31.9 | Reference range: >20
      Patient treatment journey brochure icon Patient treatment journey brochure icon Patient treatment journey brochure icon

      TDT Treatment Journey Brochure

      TDT Treatment
      Journey Brochure

      Consider walking a patient like Amina and their family through the CASGEVY TDT Treatment Journey Brochure so they can better understand each step of the treatment process and decide if CASGEVY may be right for them.

      Consider walking a patient like Amina and their family through the CASGEVY TDT Treatment Journey Brochure so they can better understand each step of the treatment process and decide if CASGEVY may be right for them.

      ALT=alanine aminotransferase; AST=aspartate aminotransferase; F=female; M=male; RBC=red blood cell; TDT=transfusion-dependent β-thalassemia.

      ALT=alanine aminotransferase; AST=aspartate aminotransferase; F=female; M=male;
      RBC=red blood cell;
      TDT=transfusion-dependent β-thalassemia.

      Person walking icon View the Patient
      Treatment Journey
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      Nearby ATCs

      IMPORTANT SAFETY INFORMATION

      IMPORTANT SAFETY INFORMATION AND INDICATION

      WARNINGS AND PRECAUTIONS

      Neutrophil Engraftment Failure

      There is potential risk of neutrophil engraftment failure after treatment with CASGEVY. In the clinical trials, all treated patients achieved neutrophil engraftment and no patients received rescue CD34+ cells.

      There is potential risk of neutrophil engraftment failure after treatment with CASGEVY. In the clinical trials, all treated patients achieved neutrophil engraftment and no patients received rescue CD34+ cells.

      INDICATION

      CASGEVY is indicated for the treatment of patients aged 12 years and older with:

      • sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs)
      • transfusion-dependent β-thalassemia (TDT)

       

      Monitor absolute neutrophil counts (ANC) and manage infections according to standard guidelines and medical judgement. In the event of neutrophil engraftment failure, patients should be infused with rescue CD34+ cells.

      Delayed Platelet Engraftment

      Delayed platelet engraftment has been observed with CASGEVY treatment. There is an increased risk of bleeding until platelet engraftment is achieved. In the clinical trials, there was no association observed between incidence of bleeding events and time to platelet engraftment.

      Monitor patients for bleeding according to standard guidelines and medical judgement. Conduct frequent platelet counts until platelet engraftment and platelet recovery are achieved. Perform blood cell count determination and other appropriate testing whenever clinical symptoms suggestive of bleeding arise.

      Hypersensitivity Reactions

      Hypersensitivity reactions, including anaphylaxis can occur due to dimethyl sulfoxide (DMSO) or dextran 40 in the cryopreservative solution. Monitor patients for hypersensitivity reactions during and after infusion.

      Off-Target Genome Editing Risk

      Although off-target genome editing was not observed in the edited CD34+ cells evaluated from healthy donors and patients, the risk of unintended, off-target editing in an individual’s CD34+ cells cannot be ruled out due to genetic variants. The clinical significance of potential off-target editing is unknown.

      ADVERSE REACTIONS

      The most common Grade 3 or 4 non-laboratory adverse reactions (occurring in ≥ 25%) were mucositis and febrile neutropenia in patients with SCD and patients with TDT, and decreased appetite in patients with SCD.

      All (100%) of the patients with TDT and SCD experienced Grade 3 or 4 neutropenia and thrombocytopenia. Other common Grade 3 or 4 laboratory abnormalities (≥ 50%) include leukopenia, anemia, and lymphopenia.

      DRUG INTERACTIONS

      No formal drug interaction studies have been performed. CASGEVY is not expected to interact with the hepatic cytochrome P450 family of enzymes or drug transporters.

      Use of Granulocyte-Colony Stimulating Factor (G-CSF): G-CSF must not be used for CD34+ HSC mobilization of patients with SCD.

      Use of Hydroxyurea: Discontinue the use of hydroxyurea at least 8 weeks prior to start of each mobilization cycle and conditioning. There is no experience of the use of hydroxyurea after CASGEVY infusion.

      Use of Voxelotor and Crizanlizumab: Discontinue the use of voxelotor and crizanlizumab at least 8 weeks prior to start of mobilization and conditioning, as their interaction potential with mobilization and myeloablative conditioning agents is not known.

      Use of Iron Chelators: Discontinue the use of iron chelators at least 7 days prior to initiation of myeloablative conditioning, due to potential interaction with the conditioning agent. Some iron chelators are myelosuppressive. If iron chelation is required, avoid the use of non-myelosuppressive iron chelators for at least 3 months and use of myelosuppressive iron chelators for at least 6 months after CASGEVY infusion. Phlebotomy can be used instead of iron chelation, when appropriate.

      USE IN SPECIFIC POPULATIONS

      Pregnancy/Lactation: CASGEVY must not be administered during pregnancy and breastfeeding should be discontinued during conditioning because of the risks associated with myeloablative conditioning. Pregnancy and breastfeeding after CASGEVY infusion should be discussed with the treating physician.

      Females and Males of Reproductive Potential: A negative serum pregnancy test must be confirmed prior to the start of each mobilization cycle and reconfirmed prior to myeloablative conditioning.

      Women of childbearing potential and men capable of fathering a child should use effective methods of contraception from start of mobilization through at least 6 months after administration of CASGEVY. Advise patients of the risks associated with conditioning agents.

      Infertility has been observed with myeloablative conditioning therefore, advise patients of fertility preservation options before treatment, if appropriate.

      INDICATION

      CASGEVY is indicated for the treatment of patients aged 12 years and older with:

      • sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs)
      • transfusion-dependent β-thalassemia (TDT)

      Please see full Prescribing Information for CASGEVY.

      Reference: 1. CASGEVY [prescribing information]. Vertex Pharmaceuticals Incorporated. Boston, MA; January 2024.