Kymriah FDA Approval, Cost, Side Effects, REMS, And Prescribing Information For The Treatment of Leukemia | M D, PharmD | RxEconsult

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Kymriah (tisagenlecleucel) FDA Approval, Cost, Side Effects, REMS, And Prescribing Information For The Treatment of Leukemia Category: Cancer by - September 12, 2017 | Views: 18466 | Likes: 0 | Comment: 0  

Kymriah approval, cost, prescribing information

Brand Name: Kymriah
Generic Name: tisagenlecleucel
Medication Class: CD19-directed genetically modified autologous T cell
Similar Drugs:  Yescarta (axicabtagene ciloleucel)
Manufacturer: Novartis
FDA Approval Date: August 2017

What is Kymriah and its mechanism of action?

Kymriah is a treatment that helps the patient’s immune system to detect and destroy leukemia cells. It is the first FDA approved cell-based gene immunotherapy which involves genetically reprogramming the patient’s T-cells with a chimeric antigen receptor T-cell (CAR-T). This reprogrammed T-cell, which is individualized to each patient, can identify and eliminate leukemia cells that express the CD19 antigen.

Each Kiymriah dose is prepared from the patient’s own T-cells. The T-cells are collected by a healthcare provider and then sent to the Novartis manufacturing center in New Jersey, where they are modified with the CAR-T. The genetically modified cells are infused back into the patient to kill the cancer cells.

What is Kymriah used for treating?

Kymriah is FDA approved for the treatment of patients 25 or under with B-cell precursor acute lymphoblastic leukemia (ALL) that is resistant to other treatments or for patients who have had relapses.

What are the cure rates for Kymriah?

The effectiveness of Kyriah was measured by levels of immature blood-forming cells (blasts) in the bone.

The cure rates and effectiveness of Kymriah were evaluated in one multicenter clinical trial. In this study 68 patients with relapsed or refractory B-cell precursor ALL were enrolled and 63 were treated. Patients received lymphodepleting chemotherapy followed by a single dose of Kymriah.

83% of patients experienced a complete remission defined as under 5% of blasts in the bone marrow, no evidence of extramedullary disease, and full recovery of blood counts within three months of treatment. The beginning of remission was between 26 and 31 days for most of the patients. 

What are the side effects of Kymriah?

The most common side effects of Kymriah include:

  • Difficulty breathing
  • Fever
  • Chills
  • Confusion
  • Nausea, vomiting, diarrhea
  • Muscle or joint pain
  • Low blood pressure
  • Dizziness/lightheadedness

Other side effects of Kymriah include:

  • Infections
  • Decreased appetite
  • Headache
  • Bleeding
  • Fast heart rate
  • Acute kidney injury
Possible serious adverse effects of Kymriah include:
  • Cytokine Release Syndrome (CRS)
  • Neurological Toxicities
  • Infections and febrile neutropenia
  • Prolonged cytopenias
  • Hypogammaglobulinemia

Kymriah is only available from specific healthcare facilities through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) because of the risk of CRS and neurological toxicity. This REMS requires healthcare facilities that would like to dispense and administer Kymriah to have the proper treatments for CRS on hand and healthcare providers that are registered and trained in the management of CRS and neurological toxicities should be available.

What is the dosage of Kymriah?

Kymriah is provided as a single-dose unit given as an Intravenous infusion under the supervision of a physician and each dose is specific to the patient.

Kymriah infusion should be preceded by a fludarabine and cyclophosphamide lymphodepleting chemotherapy.

The specific dosage of Kimriah is dependent on the patient’s weight at the time of the blood draw.

50 kg or less: 0.2-5x106 CAR-T cells/kg

Over 50kg: 0.1-2.5 x108 CAR-T cells

Kymriah should be infused 2-14 days after completion of lymphodepleting chemotherapy.

Patients should be pre-medicated with acetaminophen and diphenhydramine (or another H1-antihistamine) 30-60 minutes prior to Kymriah infusion.

How do you store Kymriah?

Kymriah comes from the manufacturer as a frozen suspension. It should be stored in the vapor phase of liquid nitrogen at less than or equal to -120 °C.

Kymriah should be thawed prior to infusion.

What are Kymriah drug interactions?

Kymriah is made using a lentivirus that shares similar genetic material to HIV. This may cause false-positive results in some HIV nucleic acid tests.

What are warnings and precautions for Kymriah?

Kymriah may cause Cytokine release syndrome (CRS). CRS is a reaction that occurs due to the release of cytokines from cells targeted by Kymriah. The cytokines are released into the circulation and cause symptoms such as high fever, low blood pressure, difficulty breathing, and possible hepatic, renal, and cardiac dysfunction. Due to the risk of CRS and Neurological toxicity, Kymriah is only available from specific healthcare facilities through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS).

Severe or life-threatening neurological toxicities may occur after taking Kymriah. The majority of these occurred within 8 weeks of infusion. The symptoms include headache, encephalopathy, dizziness, anxiety, and tremor.  Disturbances in consciousness, disorientation, confusion, agitation, seizures, and difficulty speaking may also occur. Patients should refrain from driving and operating dangerous equipment for at least 8 weeks after the infusion.

Kymriah contains dimethyl sulfoxide (DMSO) and this may cause an allergic reaction in some patients.

Kymriah may also cause serious and possibly life-threatening infections. Kymriah may cause reactivation of Hepatitis B Virus (HBV). Patients with a current or previous HBV infection should be tested for HBV antigens before starting Kymriah.

Kymriah may also cause thrombocytopenia (low platelet count) and neutropenia (low white blood cells). Thrombocytopenia increases the chance of bleeding and neutropenia increases the risk of infections.

Kymriah may also cause hypogammaglobulinemia which is an immune disorder characterized by a reduction in antibodies that help fight infections. Immunoglobulin levels should be monitored after treatment and managed with current standard guidelines.

Pregnancy: Although the effects of Kymriah on the fetus’ of pregnant women being treated have not been studied, Kymriah should be avoided in pregnant women.

Nursing: It is not known whether Kymriah is excreted in human milk or whether it affects human breast milk.

What is the cost of Kymriah?

The cost for one treatment of Kymriah is $475,000.

How to obtain prescription assistance for Kymriah?

The KYMRIAH CARES patient support program provides qualified patients with financial assistance. For more information, call KYMRIAH CARES at 1-844-459-6742.


FDA Approved Prescribing Information (PI) for Kymriah

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