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Novartis files CAR-T therapy in US, with swift review

CTL109 candidate gets FDA priority review status


Novartis has edged in front in the race to bring a CAR-T cancer immunotherapy to the US market after getting priority review status from the FDA for its CTL109 candidate.

The US regulator will now endeavour to deliver a verdict on CTL109 (tisagenlecleucel-T) within six months for its lead indication as a treatment for relapsed and refractory children and young adults with B-cell acute lymphoblastic leukemia (ALL).

Meanwhile, Novartis has confirmed it will file for approval of the cell therapy later this year in the EU, where it became one of the therapies to enter the EMA's new PRIME expedited approval pathway.

In CAR-T therapy, patients' cells are harvested and modified so they are more likely to seek out and destroy cancer cells expressing a particular antigen, in this case CD19. Once reprogrammed, the T cells are infused back into the patient in order to fight the cancer. Some companies are trying to develop off-the-shelf cells but the lead CAR-T candidates rely on cells taken from the patient.

Novartis is competing for the kudos of bringing the first of this new type of therapy to market with Kite Pharma, which filed for approval of its CAR-T candidate KTE-C19 (axicabtagene ciloleucel) as a treatment for a different indication - refractory aggressive non-Hodgkin lymphoma (NHL) in patients who are ineligible for autologous stem cell transplant (ASCT) – last December.

Behind the rivalry is a real hope that after years of development and refinement CAR-Ts could unlock a new way to fight not only haematological cancers – where the approach has yielded striking clinical results - but also eventually solid tumours if the right antigens can be identified and efficient delivery into tumour masses can be solved.

Novartis' investment in CAR-T gives the company an opportunity to catch up with rivals including Merck & Co and Bristol-Myers Squibb in the immuno-oncology market, particularly if CAR-T evolves into a platform that is safe and effective across multiple indications.

Analysts have predicted that CTL109 alone will pass the $1bn sales threshold within five years, although the CAR-T sector as a whole will be dwarfed by checkpoint inhibitors from Merck, BMS and others in that timeframe as they will require specialist delivery, and uptake is expected to be gradual.

In CTL109's pivotal ELIANA trial 82% of relapsed and refractory B-cell ALL patients who were treated with CAR-T went into complete remission, with 60% of these responders still relapse-free six months later.

Safety can be an issue with these cell therapies however, as seen with the setbacks that toppled Juno Therapeutics from its position at the head of the CAR-T race over the last few months. Nearly half (48%) of patients in ELIANA experienced grade 3 or 4 cytokine release syndrome (CRS) - a serious complication that can be life-threatening - although there were no deaths in Novartis' trial.

"The past five years have seen tremendous progress in the development and application of cellular engineering in an effort to personalise the treatment of cancer," said Carl June of the University of Pennsylvania, which originally developed CTL109 and licensed the programme to Novartis. "The results we have observed mark a potential new paradigm in the treatment of blood cancers that have not responded to standard therapies."

Article by
Phil Taylor

31st March 2017

From: Regulatory



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