The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has granted positive opinion of two chimeric antigen receptor (CAR) T-cell therapies, which use a patient’s own T cells to fight cancer.
Yescarta (axicabtagene ciloleucel) is manufactured by Kite – now a Gilead company – and indicated for the treatment of diffuse large B-cell lymphoma (DLBCL). Yescarta became the second CAR T-cell therapy to receive FDA approval in October 2017.
Kymriah (tisagenlecleucel) was the first gene modified cell therapy to be approved by the US Food and Drug Administration (FDA) in August last year. The therapy is indicated to treat acute lymphoblastic leukaemia (ALL).
According to Novartis, additional regulatory filings are under review for Kymriah in Canada, Switzerland, Australia and Japan.
The marketing authorisation recommendation is a ‘watershed moment’ for aggressive blood cancers, said Novartis Oncology CEO Liz Barrett in a statement.
"This truly transformative therapy helps address a profound unmet need, and Novartis is proud that our leadership in CAR-T innovation will make a meaningful difference to patients in the EU," she added.
The European Commission (EC), which approves medicines for use in the 28 EU Member States, as well as Norway, Iceland and Liechtenstein, will now review the therapies for regulatory approval.
While personalised gene modified cell therapies continue to claim regulatory success – earlier this year, Kymriah became the first CAR T-cell therapy to have received US regulatory approval for two distinct indications – ‘off-the-shelf’ therapies are also gathering regulatory traction.
Unlike autologous therapies, which use a patient’s own engineered T-cells, allogenic investigational therapies use engineered cells from a healthy donor for use in multiple patients.
In April this year, Allogene Therapeutics signed a collaboration agreement with Pfizer to develop allogeneic CAR T-cell therapies. The following month, French biotech Cellectis submitted an investigational new drug application to the FDA for its ‘off-the-shelf’ CAR T-cell candidate UCART22, which was approved in June.