Novartis AG (NVS – Free Report) announced that the FDA has approved its breakthrough gene transfer treatment, Kymriah (tisagenlecleucel) suspension for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.
Kymriah, formerly CTL019, is the first chimeric antigen receptor T cell (CAR-T) therapy approved. A novel immunocellular therapy and one-time treatment, Kymriah uses patient’s T cells to fight cancer.
CAR T is manufactured for individual patients using their own cells thereby making it different from typical small molecule or biologic therapies. During the treatment, T cells are drawn from a patient’s blood. These cells are then reprogrammed in the manufacturing facility to create genetically coded cells to express a chimeric antigen receptor to recognize and fight cancer cells and other B cells expressing a specific antigen.
The drug was developed in collaboration with the University of Pennsylvania.
In addition, the FDA has approved a Risk Evaluation and Mitigation Strategy (REMS) for Kymriah to inform and educate healthcare professionals about the risks that may be associated with Kymriah treatment.
Meanwhile, Novartis plans to make additional filings for Kymriah in the United Staes and EU later this year for Kymriah for the treatment of adult patients with r/r diffuse large B-cell lymphoma (DLBCL). In April, the FDA granted Breakthrough Therapy designation to Kymriah based on data from the JULIET study.
Novartis also teamed up with the United States Centers for Medicare and Medicaid Services (CMS) to improve efficiencies in current regulatory requirements to deliver value-based care and ensure access for this specific patient population.
Kymriah will be manufactured for each individual patient using their own cells at the Novartis Morris Plains, New Jersey facility.
Our Take
The approval is a major boost for Novartis given the potential in the CAR T therapy space.The approval opens up new frontiers in the treatment of cancer by advancing immunocellular therapy for children and young adults with r/r B-cell ALL which comprises approximately 25% of cancer diagnoses among children under 15 years old and is the most common childhood cancer in the United States.
