FDA to review Novartis CAR-T next month

FDA advisers will meet in July to begin their review of Novartis' CAR-T therapy tisagenlecleucel-T (CTL 109).

This will make the drug the first CAR-T candidate to be scrutinised by the FDA’s experts, and could confirm Novartis' pole position in the race to market.

Kite Pharma has been virtually neck and neck with Novartis until now, having completed its own rolling submission for its rival candidate, KTE-C19, in April.

According to a note posted by the FDA, independent experts from its Oncologic Drugs Advisory Committee (ODAC) will meet on 12 July to discuss data from CTL019.

Novartis has taken the lead over close rival Kite Pharma in the race to develop a CAR-T therapy after winning a priority review from the regulator.  The drug hasn't been given a formal 'PDUFA' date for a decision, but a ruling could come before the end of September.

If approved, analysts think Novartis’ drug could be a blockbuster worth $4.7 billion a year, while Kite’s could generate sales of $7.9 billion – making them the two most valuable orphan therapies in the pharma pipeline.

Novartis is seeking approval in relapsed/refractory B-cell acute lymphoblastic leukaemia (ALL) in children and young adults between three and 25 years of age.

Ahead of the meeting FDA staff will prepare a document outlining key information and the regulator’s thoughts on the therapy.

If approved, CAR-T therapies will be the next wave of cancer immunotherapies to hit the market, offering a powerful approach that may be able to clear some patients with hard-to-treat cancers in remission for months or years.

But there are catches – side effects can be extreme as the body’s immune system goes into overdrive to combat the cancer, and the durability of the response to treatment remains unknown.

Cytokine release syndrome (CRS) is a cascade of immune proteins that can be fatal in some cases if not managed, and has been implicated in the deaths of some patients in CAR-T trials.

Novartis's CTL109 hasn't seen any deaths in its trials, giving it a safer profile so far compared to several of its rivals.  However its pivotal ELIANA trial showed that 48% experienced grade 3 or 4 CRS, making it a common side effect which will undoubtedly raise concerns at the regulator.

Kite's candidate had enjoyed a similar profile until recently, but a belated release of news that a patient had died in a trial hit investor confidence in the company last month.