General MedicinePrescription OnlyPregnancy Cat. Not classifiedFDA Yes
Kresladi
MARNETEGRAGENE AUTOTEMCEL
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 contin...
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Brand names:KRESLADI
Quick Reference
Medicine ClassGeneral Medicine
FDA ApprovedYes
PregnancyCategory Not classified
Quick Facts
- Active ingredient
- MARNETEGRAGENE AUTOTEMCEL
- Medicine class
- General Medicine
- Brand names
- KRESLADI
- Availability
- Prescription only (Rx)
- Pregnancy category
- Not classified
- FDA approved
- Yes
What Is Kresladi?
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 contin...
Kresladi (MARNETEGRAGENE AUTOTEMCEL) belongs to the General Medicine class of medications. It was first approved by the FDA in Yes. This medication requires a prescription from a licensed healthcare provider.
Key Safety Information
- •Always follow the dose recommended by your healthcare professional. Taking more than prescribed increases the risk of side effects including serious complications.
- •Seek emergency care if you experience signs of an allergic reaction — hives, swelling, difficulty breathing, or rash. Stop the medication and call your doctor immediately.
- •Some medications can increase the risk of cardiovascular events. Tell your doctor if you have high blood pressure, a history of heart attack or stroke, or any pre-existing health condition.
- •Avoid or limit alcohol while taking Kresladi. Do not drink alcohol without first discussing it with your healthcare provider as it may increase adverse effects.
- •Take Kresladi with food or a full glass of water if stomach upset occurs. Stop use and talk to your doctor if you experience persistent stomach pain or GI symptoms.
- •Tell your doctor about all medications, supplements, and vitamins you take. Some combinations significantly increase the risk of harm.
This is a summary only. Always read the full prescribing information and consult your healthcare provider for personalized medical advice.
Uses & Indications
Kresladi is prescribed for the following conditions. Some uses are FDA-approved indications; others may be evidence-based off-label uses. Consult your healthcare provider for personalized guidance.
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 Guide
The following are general dosing guidelines only. Your actual dose should be determined by your healthcare provider based on your condition, renal/hepatic function, and other medications.
AD
Adult Dosage
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 ) 2.1 Dose 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 firs
Available Forms
Available Strengths
Side Effects
Common
- 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.
- 6.1 Clinical 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
- 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.
Rare
Medicine Interactions
Always inform your healthcare provider and pharmacist about ALL medications you take, including prescriptions, OTC medicines, vitamins, and supplements.
MajorModerateMinor
Warnings & Contraindications
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 mo
How Kresladi Works
12.1 Mechanism 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.
Pharmacokinetics
Absorption
absorption, distribution, metabolism, and elimination are not applicable
Metabolism
metabolism, and elimination are not applicable
Pregnancy & Breastfeeding Safety
Not classified
Breastfeeding
Many medications pass into breast milk in varying amounts. Before using Kresladiwhile breastfeeding, discuss the benefits and risks with your healthcare provider or pharmacist — they can weigh your dose, your infant's age, and available lactation safety data to find the safest option for you and your baby.
Storage & 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
Frequently Asked Questions
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Sources & References
- • FDA Prescribing Information — Official labeling and package insert for Kresladi
- • U.S. National Library of Medicine — MedlinePlus drug information
- • American Hospital Formulary Service (AHFS) — Clinical pharmacology monograph
- • PubMed / MEDLINE — View clinical studies
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Medical Disclaimer
The information on this page is for educational purposes only and is not intended as medical advice, diagnosis, or treatment. Always consult your doctor, pharmacist, or qualified healthcare provider before starting, stopping, or changing any medication.