FDA Approves First CAR-T Therapy to Novartis

The US Food and Drug Administration (FDA) has approved Novartis’ cancer treatment, Kymriah (tisagenlecleucel), as the first chimeric antigen receptor T cell (CAR-T) therapy available in the US. The approval was for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).

Kymriah is a customized treatment created using an individual patient’s own T-cells, a type of white blood cell known as a lymphocyte. The patient’s T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein (a CAR) that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells, according to the FDA.

Kymriah will be manufactured for each patient at Novartis’ Morris Plains, New Jersey facility. In 2012, Novartis and the University of Pennsylvania entered into a global collaboration to further research, develop and commercialize CAR-T cell therapies, including Kymriah, for the investigational treatment of cancers.

Novartis also announced a collaboration with the US Centers for Medicare and Medicaid Services (CMS) focused on improving efficiencies in current regulatory requirements in order to deliver value-based care and access for this specific patient population. This approach is intended to include indication-based pricing for medicines and supports payments for a medicine, such as Kymriah for its initial indication, based on the clinical outcomes achieved, which would eliminate inefficiencies from the healthcare system. Other value-based approaches related to future indications for Kymriah and CAR-T cell therapies are under discussion. Furthermore, Novartis is collaborating with CMS to make an outcomes-based approach available to allow for payment only when pediatric and young adult ALL patients respond to Kymriah by the end of the first month. Future potential indications would be reviewed for the most relevant outcomes-based approach.

Novartis plans additional filings for Kymriah in the US and European Union (EU) later in 2017, including applications with the FDA and European Medicines Agency (EMA), for treating adult patients with r/r diffuse large B-cell lymphoma. Additional filings beyond the US and EU are anticipated in 2018.

Kite Pharma, a Santa Monica, California-headquartered cell therapy company, is also advancing a late-stage CAR-T therapy. Kite Pharma has advanced its CAR-T therapy, axicabtagene ciloleucel, by submitting for marketing authorization to the FDA and EMA. In a separate development, Kite also submitted an investigational new drug application to the FDA to initiate a Phase I, first-in-human trial of KITE-585, another CAR-T therapy. In August 2017, Gilead Sciences agreed to acquire Kite Pharma for approximately $11.9 billion.

Source: Novartis and FDA


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