Research & Development
Novartis granted US FDA priority review for Kymriah
17 January 2018 -

The US Food and Drug Administration (FDA) has accepted Novartis' (VTX: NOVN) supplemental Biologics License Application (sBLA) for Kymriah (tisagenlecleucel), formerly CTL019, for the treatment of adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) who are ineligible for or relapse after autologous stem cell transplant (ASCT) for priority review, the company disclosed on Wednesday.

The European Medicines Agency (EMA) has also granted accelerated assessment for the Marketing Authorisation Application (MAA) for Kymriah as a treatment for children and young adults with r/r B-cell acute lymphoblastic leukaemia (ALL) and for adult patients with r/r DLBCL who are ineligible for ASCT.

Priority review and accelerated assessment are awarded to therapies that have the potential to provide a significant improvement in the safety and effectiveness of a treatment for a serious disease.

If Kymriah is approved by the US FDA and the EMA, it will be the first time a chimeric antigen receptor T cell (CAR-T) therapy has been made available for two distinct indications in non-Hodgkin lymphoma and B-cell ALL. In August 2017, the drug became the first CAR-T cell therapy to be approved by the FDA 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.

The applications to the regulatory bodies in the US and the EU were based on data from the Novartis-sponsored global clinical trial programme of Kymriah in children and young adults with r/r B-cell ALL and adult patients with r/r DLBCL, which demonstrated the efficacy and safety of Kymriah across studies, as well as the results from the pivotal Phase II JULIET clinical trial.

This year, the company is also planning further regulatory submissions for Kymriah in paediatric and young adult patients with r/r B-cell ALL and adult patients with r/r DLBCL beyond the US and EU.