What is KYMRIAH or tisagenlecleucel?
KYMRIAH is the trade name of tisagenlecleucel, manufactured by Novartis Pharmaceuticals. KYMRIAH, a cell-based gene therapy, is approved in the United States for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse – As per the US FDA. This happens to be the first cell-based gene therapy approved by the US FDA. To be specific, KYMRIAH is a (CAR) T-cell therapy. The entire therapy is innovated based on the possibility of genetically reprogramming patients own cells, to ultimately attack the cancer cells.
KYMRIAH – FDA Approved Cellular and Gene Therapy Product
KYMRIAH was granted its US FDA approval in August 2017. This approval was based on a multicenter clinical trial involving 63 patients. These are pediatric and young adult patients relapsed or refractory B-cell precursor ALL. The study outcomes highlight an 83% overall remission rate within three months. Based on the safety and efficacy data, KYMRIAH was granted Breakthrough Therapy designation. More information can be found in the US FDA Press Release. In May 2018, KYMRIAH was given the US FDA approval for treating adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma.
How does treatment with KYMRIAH (tisagenlecleucel) work?
Cellular and Gene Therapies are different and advanced in comparison with Chemotherapy and Immunotherapy. Unlike drugs that are administered in regular clinical settings, some of them have to be personalized. KYMRIAH is a novel solution that involves the reprogramming of patient’s White Blood Cells. Through a process called “Leukapheresis”, WBCs are isolated and extracted. These blood cells are used to make KYMRIAH at the company’s manufacturing facility. Once the genetically modified solution is prepared, KYMRIAH will be sent to the Healthcare facility for administration. It would require less than one hour for the infusion. In case of Pediatric and Young Adult B-cell ALL treatment, a single dose of KYMRIAH contains 0.2 to 5.0 x 106 CAR-positive viable T cells per kg of body weight for patients 50 kg or less, or 0.1 to 2.5 x 108 CAR-positive viable T cells for patients more than 50 kg, suspended in one to three patient-specific infusion bag(s) for IV infusion. A single dose of KYMRIAH contains 0.6 to 6.0 x 108 CAR-positive viable T cells suspended in one to three patient-specific infusion bag(s) for IV infusion is meant for Adult Relapsed or Refractory Diffuse Large B-cell Lymphoma treatment. KYMRIAH is supplied as a frozen suspension of genetically modified autologous T cells in an infusion bag(s) labeled for the specific recipient. It is shipped in a liquid nitrogen Dewar. Product and patient-specific labels are located inside the Dewar. Refer to the KYMRIAH Prescribing Information for more specific details.
Patients and caregivers can visit www.kymriah.com for comprehensive information. The website provides avenues to find a treatment center and option to register. This is available for US patients at present.
KYMRIAH in India – Is there a possibility?
Right now there is limited information about KYMRIAH India. The active presence of Novartis in India would certainly provide reasons for Indian patients to be hopeful about the future prospects. More information is likely to emerge in the days to come. You can write to us at nhs@noelgenomics.com to express your interest. We are committed to provide authentic access information and assist Indian patients with access to advanced therapeutic solutions.
