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Date: 2017-07-31

Type of information: Submission of a Market Application in the EU

Product name: axicabtagene ciloleucel (KTE-C19)

Compound: autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor

Therapeutic area: Cancer - Oncology - Rare diseases

Action mechanism: cell therapy/immunotherapy product/CAR-T cell therapy. Axicabtagene ciloleucel (KTE-C19) is Kite Pharma's lead product candidate in which a patient's T cells are genetically modified using a gammaretroviral vector to express a chimeric antigen receptor (CAR) designed to target the antigen CD19, a protein expressed on the cell surface of B cell lymphomas and leukemias. Axicabtagene ciloleucel (KTE-C19) received Breakthrough Therapy Designation (BTD) by the FDA in December 2015 .

Company: Kite Pharma (USA - CA)

Disease: relapsed/refractory aggressive B-cell non-Hodgkin lymphoma (NHL) who are ineligible for autologous stem cell transplant (ASCT), diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL)

Latest news:

  • • On July 31, 2017, Kite Pharma announced that it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for axicabtagene ciloleucel as a treatment for patients with relapsed/refractory diffuse large B-cell lymphoma, transformed follicular lymphoma, and primary mediastinal B-cell lymphoma who are ineligible for autologous stem cell transplant. This application represents the first chimeric antigen receptor (CAR) T-cell therapy submitted to the EMA. The MAA for axicabtagene ciloleucel is supported by data from the ZUMA-1  trial. In May 2016, the CHMP and CAT granted access to its newly established Priority Medicines (PRIME) regulatory initiative for axicabtagene ciloleucel in the treatment of patients with refractory DLBCL.
  • • On May 26, 2017, Kite Pharma announced that the FDA has accepted for priority review the Biologics License Application (BLA) for axicabtagene ciloleucel. The submission follows positive data demonstrated with a single infusion of axicabtagene ciloleucel in the ZUMA-1 Phase 2 trial in patients with refractory aggressive non-Hodgkin lymphoma (NHL). The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of November 29, 2017. The filing acceptance is supported by data from the ZUMA-1 Phase 2 trial which met the primary endpoint of objective response rate recorded after a single infusion of axicabtagene ciloleucel with 82 percent (p < 0.0001). At a median follow-up of 8.7 months, 44 percent of patients were in ongoing response, which included 39 percent of patients in complete response.
  • The most common grade 3 or higher adverse events included anemia (43 percent), neutropenia (39 percent), decreased neutrophil count (32 percent), febrile neutropenia (31 percent), decreased white blood cell count (29 percent), thrombocytopenia (24 percent), encephalopathy (21 percent) and decreased lymphocyte count (20 percent). There were three deaths throughout the course of the registrational trial not due to disease progression, of which two events, were deemed related to axicabtagene ciloleucel.
  • • On March 31, 2017,  Kite Pharma announced that it has completed the rolling submission with the FDA of the Biologics License Application (BLA) for axicabtagene ciloleucel as a treatment for patients with relapsed or refractory aggressive non-Hodgkin lymphoma (NHL) who are ineligible for autologous stem cell transplant (ASCT). If approved, Kite plans to commercially launch axicabtagene ciloleucel in 2017. Kite is also planning a regulatory submission to the European Medicines Agency (EMA) for axicabtagene ciloleucel in 2017.
  • • On December 5, 2016, Kite Pharma announced that it has initiated the rolling submission with the FDA of the Biologics License Application (BLA) for KTE-C19 as a treatment for patients with relapsed/refractory aggressive B-cell non-Hodgkin lymphoma (NHL) who are ineligible for autologous stem cell transplant (ASCT). The pivotal ZUMA-1 study supporting this submission enrolled patients with chemorefractory diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL), three subtypes of aggressive NHL . The company expects to complete its BLA submission by the end of the first quarter of 2017. Kite also announced that the United States Adopted Name, or USAN, for KTE-C19 will be axicabtagene ciloleucel. If approved, Kite plans to commercially launch KTE-C19 in 2017. Kite is also planning a regulatory submission to the European Medicines Agency (EMA) for axicabtagene ciloleucel in 2017. Kite was granted access to Priority Medicines (PRIME) regulatory support in 2016 by the EMA for axicabtagene ciloleucel (KTE-C19) for the treatment of refractory DLBCL.
  • •  On April 25, 2016, the FDA has granted orphan drug designation for autologous T-cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (KTE-C19) for treatment of follicular lymphoma.
  • •  On December 7, 2015, Kite Pharma announced that the FDA has granted Breakthrough Therapy Designation status to KTE-C19, for the treatment of patients with refractory diffuse large B cell lymphoma (DLBCL), primary mediastinal B cell lymphoma (PMBCL), and transformed follicular lymphoma (TFL). Four clinical trials are currently ongoing: ZUMA-1ZUMA-2ZUMA-3 and ZUMA-4.
  • • On October 6-8, 2015, the Committee for Orphan Medicinal Products (COMP) has recommended the granting of an orphan designation for autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor for treatment of follicular lymphoma.
  • • On November 11-13, 2014, the Committee for Orphan Medicinal Products (COMP) has recommended the granting of an orphan designation for autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor for treatment of diffuse large B cell lymphoma.

Patents:

Submission of marketing authorization application USA : 2017-03-31

Submission of marketing authorization application UE: 2017-07-31

Withdrawal of marketing authorization application USA:

Withdrawal of marketing authorization application UE:

US authorization:

UE authorization:

Favourable opinion UE:

Favourable opinion USA:

Orphan status USA: 2016-04-25

Orphan status UE: 2015-11-11/2014-12-16

Pediatric exclusivit _USA:

Pediatric exclusivity UE:

OTC status:

Other news:

Is general: Yes