KRESLADI MARNETEGRAGENE AUTOTEMCEL 6100000 1/mL Injection, Suspension, 30 mL
🆔 Identity & classification
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🏭 Manufacturer & labeler
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🩺 Clinical
Marnetegragene autotemcel injection is used to treat leukocyte adhesion deficiency-I (LAD-I; an inherited condition in which the body makes immune cells that cannot fight infections). Marnetegragene autotemcel is in a class of medications called autologous cellular immunotherapy, a type of medication prepared using cells from the patient's own blood. It works by helping your body make certain immune cells to help fight infections.
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🧪 Inactive Ingredients / Excipients
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💲 Pricing
A drug doesn't have one price. Each row is a different public payment system, and none is what you'd pay at the counter — that depends on your insurance. The ⓘ on each row explains what it measures.
| Price system | Per mL | Per package |
|---|---|---|
| Retail pharmacies payNADAC · weekly | Not in the retail survey — common for institutional, discontinued, or low-volume packs. | |
| Medicaid paysCMS SDUD · 12 mo | No recent Medicaid claims on file for this NDC — rare and low-volume NDCs are suppressed in the public data. | |
| Medicare drug plans payPart D · quarterly | No Part D plan price is available for this NDC in our data. | |
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🔁 Therapeutic equivalents
| Product | Labeler | Pack | NADAC/unit | TE | Status | Price vs. this |
|---|---|---|---|---|---|---|
| Kresladi 6100000 1/mLthis 83537-0034-01 | Rocket | 30 ml | — | — | FDA listed | — |
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⏳ Availability & biosimilar status
Biologics have no small-molecule generics; biosimilar competition is tracked in the FDA Purple Book.
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📦 Packaging — all sizes for this product
| Package NDC | Description | Marketing start | Status |
|---|---|---|---|
| 83537-0034-01 You're viewing this | 30 mL in 1 BAG (83537-034-01) | 2026-03-26 | Active |
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| NDC identity (package / product / labeler codes) | ✓ Available |
| Labeler | ✓ Available |
| Product & package description | ✓ Available |
| Marketing category & status | ✓ Available |
| Active ingredient / dosage form / route | ✓ Available |
| FDA label (SPL via DailyMed) | ✓ Available |
| Package photos | ✓ Available |
| Inactive ingredients (structured) | — Not published for this NDC The labeler did not submit a structured excipient list, or no SPL is available. |
| NADAC pharmacy acquisition price (CMS) | — Not published for this NDC CMS publishes NADAC only for NDCs reported in its retail-pharmacy survey. |
| Orange Book / therapeutic-equivalence data | — Not published for this NDC Applies only to products approved under an NDA/ANDA; many listings are out of scope. |
| HCPCS J-code billing crosswalk | — Not published for this NDC Most self-administered / retail products have no J-code — that is normal. |
| Medicaid utilization (CMS SDUD) | — Not published for this NDC CMS reports utilization only for NDCs with Medicaid claims above its privacy threshold. |
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📄 Full FDA label FDA SPL
🎯 Indications and Usage ▾
1 INDICATIONS AND USAGE KRESLADI is indicated for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen (HLA)-matched sibling donor for allogeneic hematopoietic stem cell transplant. This indication is approved under accelerated approval based on increase in neutrophil CD18 and CD11a surface expression [see Clinical Studies (14) ]. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
KRESLADI is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen (HLA)-matched sibling donor for allogeneic hematopoietic stem cell transplant. ( 1 ) This indication is approved under accelerated approval based on increase in neutrophil CD18 and CD11a surface expression [see Clinical Studies (14) ] . Continued approval may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
⏱️ Dosage and Administration ▾
2 DOSAGE AND ADMINISTRATION For autologous use only. For intravenous use only. Patients must undergo hematopoietic stem cell (HSC) mobilization followed by apheresis to obtain CD34+ cells for KRESLADI manufacturing.
( 2.2 ) Dosing of KRESLADI is based on the number of CD34+ cells in the infusion bag(s) per kg of body weight. ( 2.1 ) The minimum recommended dose is 2.8 × 10 6 CD34+ cells/kg. ( 2.1 ) Full myeloablative conditioning must be administered before infusion of KRESLADI.
( 2.2 ) Verify the patient's identity matches the unique patient identification information on the KRESLADI infusion bag(s) prior to infusion. ( 2.2 ) Do not sample, alter, or irradiate KRESLADI. ( 2.2 ) Do not use an in-line blood filter or infusion pump.
( 2.3 )
2.1Dose For autologous use only. For one-time single-dose intravenous use only. The recommended minimum dose of KRESLADI is a single intravenous infusion of 2.8 × 10 6 CD34+ cells/kg. The dose is calculated based on the patient's weight prior to first apheresis. See the Lot Information Sheet provided with the product shipment for additional information pertaining to dose. Up to two drug product lots may be administered to meet the minimum dose.
2.2Preparation Before KRESLADI Infusion Before mobilization, apheresis, and conditioning are initiated, confirm that autologous hematopoietic stem cell (HSC) transplantation is appropriate for the patient. Consider administering ustekinumab prior to mobilization and apheresis and/or prior to KRESLADI infusion. Mobilization and Apheresis Administer granulocyte-colony-stimulating factor (G-CSF) and plerixafor for HSC mobilization followed by apheresis to obtain CD34+ cells for product manufacturing.
Weigh the patient prior to the first apheresis collection. Collect a recommended target number of CD34+ cells of 20 × 10 6 CD34+ cells/kg. Repeat additional cycles of mobilization and apheresis after at least 14 days to obtain more cells for KRESLADI manufacture if the minimum dose of 2.8 × 10 6 CD34+ cells/kg is not met.
Back-Up CD34+ Cell Collection Obtain and cryopreserve an additional collection of CD34+ cells of ≥ 2 × 10 6 CD34+ cells/kg at the treatment center prior to initiating myeloablative conditioning and infusion with KRESLADI. Administer the back-up collection for rescue treatment if there is: 1) compromise of KRESLADI after initiation of conditioning and before KRESLADI infusion, 2) primary engraftment failure, or 3) loss of engraftment after infusion with KRESLADI. Myeloablative Conditioning Do not begin conditioning until KRESLADI has been received and stored at the treatment center and the availability of the back-up collection of CD34+ cells is confirmed.
Administer myeloablative conditioning before infusion of KRESLADI based on institutional guidelines. Consult the prescribing information for the conditioning agents prior to treatment. Infuse KRESLADI 24-48 hours after completion of myeloablative conditioning.
Receipt and Storage of KRESLADI Ensure the availability of vapor phase of liquid nitrogen storage at less than or equal to -150°C (-238°F) at the treatment center. KRESLADI is shipped to the treatment center in the vapor phase of liquid nitrogen shipper. Confirm the patient's identity with the patient identifiers on the shipper and labels.
If there are any concerns about the product or packaging upon receipt, contact Rocket Pharmaceuticals at 1-800-982-2410. Keep the infusion bag(s) in the metal cassette(s) and transfer KRESLADI from the vapor phase of liquid nitrogen shipper to the onsite vapor phase of liquid nitrogen storage (at ≤ -150°C (≤ -238°F) until ready for thaw and administration). Preparation of KRESLADI for infusion Confirm the infusion time in advance and adjust the start time of KRESLADI thaw such that it will be available for infusion when the patient and the healthcare provider are ready.
Complete infusion of each bag of KRESLADI within 30 minutes of the start of thaw. 1. Confirm patient identity: Prior to KRESLADI…
💊 Dosage Forms and Strengths ▾
3 DOSAGE FORMS AND STRENGTHS KRESLADI is a cell suspension for intravenous infusion. KRESLADI is composed of one or two infusion bags which contain 0.34 to 6.1 × 10 6 cells/mL (including 0.32 to 6.1 × 10 6 CD34+ cells/mL) suspended in a cryopreservation solution [see How Supplied/Storage and Handling (16) ]. Each infusion bag contains approximately 30 mL of KRESLADI.
Up to two drug product lots may be administered to meet the recommended dose. See the Lot Information Sheet for actual strength and dose. KRESLADI is a cell suspension for intravenous infusion.
( 3 ) KRESLADI is composed of one or two infusion bags which contain 0.34 to 6.1 × 10 6 cells/mL (including 0.32 to 6.1 × 10 6 CD34+ cells/mL) suspended in a cryopreservation solution. ( 3 ) Each infusion bag contains approximately 30 mL of KRESLADI. ( 3 )
⛔ Contraindications ▾
4 CONTRAINDICATIONS None. None. ( 4 )
⚠️ Warnings and Cautions ▾
5 WARNINGS AND PRECAUTIONS Serious Infections: Monitor patients for signs and symptoms of infection before and after KRESLADI infusion and treat appropriately. Administer prophylactic antimicrobials according to institutional guidelines. ( 5.1 ) Veno-Occlusive Disease: Monitor patients for signs and symptoms of veno-occlusive disease including assessment of liver function tests during the first month following KRESLADI infusion.
( 5.2 ) Neutrophil Engraftment Failure: Monitor absolute neutrophil counts (ANC) after KRESLADI infusion. If neutrophil engraftment does not occur administer rescue cells. ( 5.3 ) Delayed Platelet Engraftment: Monitor patients for thrombocytopenia and bleeding until platelet engraftment and count recovery.
( 5.4 ) LVV-mediated Insertional Oncogenesis: Monitor patients at least annually for hematologic malignancies for at least 15 years after KRESLADI infusion. ( 5.5 ) Hypersensitivity Reactions: Monitor for hypersensitivity reactions during the infusion. ( 5.6 )
5.1Serious Infections Serious infections have occurred with KRESLADI administration [see Adverse Reactions (6.1) ]. Increased susceptibility to infections may occur due to administration of myeloablative conditioning prior to KRESLADI infusion. Monitor patients for signs and symptoms of infection before and after KRESLADI infusion and treat appropriately.
Administer prophylactic antimicrobials according to institutional guidelines. Avoid administration of KRESLADI in patients with active bloodstream infections or other serious, untreated infections. Any blood products required after KRESLADI infusion should be irradiated.
5.2Veno-Occlusive Disease Veno-occlusive disease has occurred with KRESLADI treatment [see Adverse Reactions (6.1) ]. Increased susceptibility to veno-occlusive disease may occur due to administration of myeloablative conditioning prior to KRESLADI infusion. Monitor patients for signs and symptoms of veno-occlusive disease including assessment of liver function tests during the first month following KRESLADI infusion.
5.3Neutrophil Engraftment Failure Neutrophil engraftment failure may occur after treatment with KRESLADI. Neutrophil engraftment failure is defined as failure to achieve three consecutive absolute neutrophil counts (ANC) ≥ 500 cells/microliter obtained on different days by Day 43 after infusion of KRESLADI. Monitor neutrophil counts until engraftment has been achieved.
If neutrophil engraftment failure occurs in a patient treated with KRESLADI, provide rescue treatment with the back-up collection of CD34+ cells [see Preparation Before KRESLADI Infusion (2.2) ] .
5.4Delayed Platelet Engraftment Delayed platelet engraftment may occur after treatment with KRESLADI. Monitor platelet counts and bleeding until platelet engraftment and platelet recovery are achieved.
5.5LVV-Mediated Insertional Oncogenesis Lentiviral vector (LVV)-mediated insertional oncogenesis may occur after treatment with KRESLADI. Hematologic malignancy is a lifelong risk and patients treated with KRESLADI may develop hematologic malignancy at any time following treatment. Monitor for hematologic malignancies clinically, and with a complete blood count (with differential) at least annually and integration site analysis as warranted for at least 15 years after treatment with KRESLADI and as clinically indicated.
If malignancy is detected in any patient who received KRESLADI, contact Rocket Pharmaceuticals, Inc. at 1-800-982-2410 for reporting and to obtain instructions on collection of samples for testing.
5.6Hypersensitivity Reactions Hypersensitivity reactions including anaphylaxis may occur with the infusion of KRESLADI. The dimethyl sulfoxide (DMSO) in KRESLADI may cause hypersensitivity reactions which may occur in patients with and without prior exposure to DMSO. Monitor patients for signs and symptoms of hypersensitivity reactions during and after KRESLADI infusion. If a hypersensitivity reaction occurs, pause infusion if ongoing and ma…
🤒 Adverse Reactions ▾
6 ADVERSE REACTIONS The most common non-laboratory adverse reactions (≥ 30%): mucositis, upper respiratory tract infection, viral infection, febrile neutropenia, skin lesion, nausea/vomiting, rash/dermatitis, pyrexia, device related infection, and skin infection. ( 6.1 ) The most common laboratory adverse reactions (≥ 30%): hemoglobin decreased, platelet count decreased, neutrophil count decreased, leukocyte count decreased, aspartate aminotransferase increased, and alanine aminotransferase increased. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Rocket Pharmaceuticals at 1-800-982-2410 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
6.1Clinical Trials Experience As clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety data described in this section reflect exposure to KRESLADI in one clinical study (Study RP-L201-0318). A total of 9 pediatric patients with severe LAD-I received a single dose of intravenous KRESLADI with a median dose of 4.3 × 10 6 CD34+ cells/kg (range: 2.8 to 10 CD34+ cells/kg) [see Clinical Studies (14) ].
The median duration of follow up after KRESLADI administration was 4.2 years (range: 3.6 to 5.7 years). Serious adverse reactions were reported in 4 patients (44%) including serious infections (n=4), pulmonary arterial hypertension (n=1), sensorineural deafness (n=1), and veno-occlusive disease (n=1). Table 1 presents the most common adverse reactions reported in Study RP-L201-0318.
Table 1: Non-Laboratory Adverse Reactions Reported in ≥ 20% of Patients in Study RP-L201-0318 (N=9) Adverse Reaction All Grades (%) Grade ≥3 (%) Gastrointestinal disorders Note: Adverse reactions are defined as adverse events that occurred from myeloablative conditioning administration through year 2 following KRESLADI administration. Nausea/Vomiting Includes multiple related terms 4 (44%) 0 Constipation 2 (22%) 0 General disorders and administration site conditions Mucositis 8 (89%) 5 (56%) Febrile neutropenia 6 (67%) 6 (67%) Pyrexia 3 (33%) 0 Infections and infestations Upper respiratory tract infection 8 (89%) 5 (56%) Viral infection 7 (78%) 1 (11%) Skin infection 3 (33%) 0 Device-related infection Device-related infection includes vascular device infection, device related bacteremia, and bacteremia that occurred with a central line in place 3 (33%) 2 (22%) Lower respiratory tract infection 2 (22%) 1 (11%) Gastroenteritis 2 (22%) 1 (11%) Skin candidiasis 2 (22%) 0 Urinary tract infection 2 (22%) 0 Skin and subcutaneous tissue disorders Skin lesion Skin lesion includes pyoderma gangrenosum, skin lesion, aseptic pustule, lip erythema, hand erythema, skin erythema at port site, and skin hyperpigmentation 6 (67%) 1 (11%) Rash Rash includes rash, eczema, atopic dermatitis and diaper dermatitis 4 (44%) 0 Alopecia 2 (22%) 0 Table 2 presents laboratory abnormalities that worsened from baseline in ≥20% of patients in Study RP-L201-0318.
Table 2: Laboratory Abnormalities that Worsened from Baseline Reported in ≥ 20% of Patients in Study RP-L201-0318 (N=9) Baseline laboratory values were assessed prior to myeloablative conditioning Laboratory Abnormality All events occurred within 30 days post-infusion except for liver enzymes increased, which occurred in 3 patients within 30 days and in one patient after 90 days post-infusion All Grades (%) Grade ≥ 3 (%) Hemoglobin decreased 9 (100%) 9 (100%) Platelet count decreased 9 (100%) 9 (100%) Neutrophil count decreased 9 (100%) 9 (100%) Leukocyte count decreased 5 (56%) 5 (56%) Aspartate aminotransferase increased 4 (44%) 0 Alanine aminotransferase increased 3 (33%) 0
🔄 Drug Interactions ▾
7 DRUG INTERACTIONS No formal drug interaction studies have been performed. KRESLADI is not expected to interact with the hepatic cytochrome P-450 family of enzymes or drug transporters. Anti-retrovirals : Patients should not take anti-retroviral medications for one month prior to mobilization, or for the expected duration required for elimination of the anti-retroviral medications, and until all cycles of apheresis are completed. ( 7.2 )
7.1Vaccines The safety and effectiveness of immunization with live viral vaccines during or following KRESLADI treatment has not been studied. Vaccination is not recommended during the 6 weeks preceding the start of myeloablative conditioning, and until hematological recovery following treatment with KRESLADI. Where feasible, administer childhood vaccinations prior to myeloablative conditioning for KRESLADI.
7.2Anti-retroviral Use Patients should not take anti-retroviral medications for at least one month prior to initiating medications for stem cell mobilization and for the expected duration for elimination of the medications, and until all cycles of apheresis are completed [see Warnings and Precautions (5.6) ] . Anti-retroviral medications may interfere with manufacturing of KRESLADI.
👥 Use in Specific Populations ▾
8 USE IN SPECIFIC POPULATIONS
8.1Pregnancy Risk Summary There are no available data from KRESLADI administration in pregnant women. No animal reproductive and developmental toxicity studies have been conducted to assess whether KRESLADI can cause fetal harm when administered to a pregnant woman. No nonclinical germline transmission studies have been conducted with KRESLADI.
KRESLADI must not be administered during pregnancy because of the risk associated with conditioning. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively.
8.2Lactation Risk Summary There is no available data regarding the presence of KRESLADI in human milk, the effect on the breastfed infant, and the effects on milk production. Because of the potential risks associated with myeloablative conditioning, discontinue breastfeeding during conditioning. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for KRESLADI and any potential adverse effects on the breastfed child from KRESLADI or from the underlying maternal condition.
8.3Females and Males of Reproductive Potential Pregnancy Testing A negative serum pregnancy test must be confirmed prior to the start of mobilization and re-confirmed prior to conditioning procedures and before administration of KRESLADI in females of childbearing potential. Contraception There are insufficient exposure data to provide a precise recommendation on duration of contraception following treatment with KRESLADI. Consult the Prescribing Information of the mobilization and conditioning agents for information on the need for effective contraception.
Infertility There are no data on the effects of KRESLADI on fertility. Data are available on the risk of infertility with myeloablative conditioning. Advise patients of the option to cryopreserve semen or ova before treatment, if appropriate.
8.4Pediatric Use The safety and efficacy of KRESLADI have been established in pediatric patients with severe LAD-I. The use of KRESLADI in the pediatric population was supported by one clinical study which treated 9 patients 9.8 months to 9.8 years of age [see Adverse Reactions (6) and Clinical Studies (14) ] .
8.5Geriatric Use The safety and effectiveness of KRESLADI has not been established in geriatric patients. Clinical studies of KRESLADI for this indication did not include patients aged 65 years and over.
8.6Patients Seropositive for Human Immunodeficiency Virus (HIV) KRESLADI has not been studied in patients with HIV-1, HIV-2, HTLV-1, or HTLV-2. A negative serology test for HIV is necessary to ensure acceptance of apheresis material for KRESLADI manufacturing. Apheresis material from patients with a positive test for HIV will not be accepted for KRESLADI manufacturing.
🤰 Pregnancy ▾
8.1Pregnancy Risk Summary There are no available data from KRESLADI administration in pregnant women. No animal reproductive and developmental toxicity studies have been conducted to assess whether KRESLADI can cause fetal harm when administered to a pregnant woman. No nonclinical germline transmission studies have been conducted with KRESLADI.
KRESLADI must not be administered during pregnancy because of the risk associated with conditioning. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively.
🧒 Pediatric Use ▾
8.4Pediatric Use The safety and efficacy of KRESLADI have been established in pediatric patients with severe LAD-I. The use of KRESLADI in the pediatric population was supported by one clinical study which treated 9 patients 9.8 months to 9.8 years of age [see Adverse Reactions (6) and Clinical Studies (14) ] .
🧓 Geriatric Use ▾
8.5Geriatric Use The safety and effectiveness of KRESLADI has not been established in geriatric patients. Clinical studies of KRESLADI for this indication did not include patients aged 65 years and over.
🧬 Clinical Pharmacology ▾
12 CLINICAL PHARMACOLOGY
12.1Mechanism of Action KRESLADI adds functional copies of the ITGB2 gene into patients' hematopoietic stem cells (HSCs) through transduction of autologous CD34+ cells with LV-RP-201. After KRESLADI infusion, transduced CD34+ HSCs engraft in the bone marrow and differentiate into various cell types, including leukocytes capable of expressing functional CD18 protein. Functional CD18 protein enables formation of the CD18/CD11a heterodimer (Leukocyte Function-Associated Antigen-1, LFA-1) which facilitates leukocyte adhesion to endothelial surfaces and extravasation to infectious and inflammatory sites.
12.2Pharmacodynamics In Study RP-L201-0318, Neutrophil CD18 and CD11a surface expression after KRESLADI administration were evaluated [see Clinical Studies (14) ] .
12.3Pharmacokinetics The nature of KRESLADI is such that conventional studies on pharmacokinetics, absorption, distribution, metabolism, and elimination are not applicable.
🧬 Mechanism of Action ▾
12.1Mechanism of Action KRESLADI adds functional copies of the ITGB2 gene into patients' hematopoietic stem cells (HSCs) through transduction of autologous CD34+ cells with LV-RP-201. After KRESLADI infusion, transduced CD34+ HSCs engraft in the bone marrow and differentiate into various cell types, including leukocytes capable of expressing functional CD18 protein. Functional CD18 protein enables formation of the CD18/CD11a heterodimer (Leukocyte Function-Associated Antigen-1, LFA-1) which facilitates leukocyte adhesion to endothelial surfaces and extravasation to infectious and inflammatory sites.
📦 How Supplied / Storage and Handling ▾
16 HOW SUPPLIED/STORAGE AND HANDLING KRESLADI is supplied in one or two infusion bags containing a frozen suspension of genetically modified autologous cells enriched for CD34+ cells in a cryo-preservative medium containing 5% DMSO and dextran 40. Each infusion bag contains approximately 30 mL and is individually packed within an overwrap in a metal cassette for protection. KRESLADI is shipped from the manufacturing facility to the infusion center in a cryoshipper, which may contain one or two cassettes intended for treatment of a single patient.
A Lot Information Sheet is affixed inside the shipper. 50 mL infusion bag and metal cassette (NDC 83537-034-01) Match the identity of the patient with the patient identifiers on the cassette(s) and Lot Information Sheet upon receipt. Keep the infusion bag(s) in the cassette(s) and store KRESLADI frozen in the vapor phase of liquid nitrogen at less than or equal to -150°C (≤ -238°F) until ready for thaw and administration.
Thaw KRESLADI prior to infusion [see Dosage and Administration (2) ]. Do not re-freeze after thawing. Do not irradiate KRESLADI, as this could lead to inactivation.
📦 Storage and Handling ▾
Keep the infusion bag(s) in the cassette(s) and store KRESLADI frozen in the vapor phase of liquid nitrogen at less than or equal to -150°C (≤ -238°F) until ready for thaw and administration. Thaw KRESLADI prior to infusion [see Dosage and Administration (2) ]. Do not re-freeze after thawing. Do not irradiate KRESLADI, as this could lead to inactivation.
📋 Description ▾
11 DESCRIPTION KRESLADI (marnetegragene autotemcel) is an autologous hematopoietic stem cell (HSC)-based gene therapy prepared from the patient's HSCs, which are collected via apheresis procedure(s). The autologous cells are enriched for CD34+ cells, then transduced ex vivo with LV-RP-201 overnight in the presence of defined growth factors before harvest and suspended in a cryopreservation solution. LV-RP-201 is a replication-incompetent, self-inactivating lentivirus vector encoding for the CD18 β-subunit of human β2 integrins ( ITGB2 ).
The ITGB2 gene is under the control of a human chimeric promoter (a fusion of the FES and CTSG minimal 5'-flanking regions). The transduced CD34+ cells are washed, formulated into a suspension, and then cryopreserved. KRESLADI is frozen in a patient-specific infusion bag(s) and is thawed prior to intravenous administration [see Dosage and Administration (2.1) , How Supplied/Storage and Handling (16) ].
The thawed product is a colorless to slightly white suspension of cells. Due to the presence of cells, the solution may be clear to slightly cloudy and may contain visible cell aggregates. The formulation contains 5% (v/v) dimethyl sulfoxide (DMSO) and dextran.
💬 Information for Patients ▾
17 PATIENT COUNSELING INFORMATION Manufacturing Failure Ensure that the patient and/or caregiver understand the risk of manufacturing failure. In case of manufacturing failure or the need for additional cells, additional cell collection and manufacturing of KRESLADI would be needed [see Dosage and Administration (2.2) ]. Serious Infections Inform the patient and/or caregiver that patients are at increased risk of developing a variety of infections, which may be due to administration of myeloablative conditioning prior to treatment with KRESLADI.
Advise the patient and/or caregiver to contact their healthcare provider if the patient develops any symptoms of infection such as fever, sweats or chills, cough or flu-like symptoms, muscle aches, or warm, red, painful areas on the skin [see Warnings and Precautions (5.1) ]. Veno-Occlusive Disease Inform the patient and/or caregiver of the risk of veno-occlusive disease, which may be due to administration of myeloablative conditioning prior to treatment with KRESLADI. Advise the patient and/or caregiver of the plan to monitor liver function following treatment with KRESLADI [see Warnings and Precautions (5.2) ].
Neutrophil Engraftment Failure Inform the patient and/or caregiver of the potential risk of neutrophil engraftment failure and the need for rescue treatment with their back-up collection of CD34+ cells, if engraftment failure occurs [see Warnings and Precautions (5.3) ]. Delayed Platelet Engraftment Inform the patient that a risk of bleeding or bruising exists after myeloablative conditioning and before platelet engraftment occurs [see Warnings and Precautions (5.4) ]. LVV-mediated insertional oncogenesis Inform the patient and/or caregiver that the risk for insertional oncogenesis is lifelong and the patient will need monitoring for hematologic malignancies via blood tests at least annually for at least 15 years after treatment with KRESLADI.
Advise patients that they may require more frequent blood tests and invasive bone marrow biopsies if routine blood tests are concerning for the development of malignancy. Advise patients to have their treating physician contact Rocket Pharmaceuticals, Inc. at 1-800-982-2410 if they are diagnosed with a malignancy [see Warnings and Precautions (5.5) ]. Hypersensitivity Reactions Inform patients to seek immediate medical attention for signs or symptoms associated with hypersensitivity reactions [see Warnings and Precautions (5.6) ].
Laboratory Test Interference Advise patients that they may test positive for HIV if tested using a PCR assay after being treated with KRESLADI [see Warnings and Precautions (5.8) ]. Advise the patient and/or caregiver that they should notify any healthcare provider about this possibility prior to being tested for HIV. Blood, Organ, Tissue and Cell Donation Inform patients that they should not donate blood, organs, tissues, or cells at any time in the future [see Dosage and Administration (2.3) , Warnings and Precautions (5.9) ].