NICE backs NHS use of Autolus' leukaemia CAR-T

Hundreds of people with a rare and aggressive form of leukaemia will be able to access a CAR-T therapy developed by UK biotech Autolus after it was recommended for routine commissioning by the NHS.

Reimbursement authority NICE has ruled that London-based Autolus' Aucatzyl (obecabtagene autoleucel or obe-cel) can be used by the NHS in England and Wales to treat adults with relapsed or refractory B-cell precursor acute lymphoblastic leukaemia (ALL), though only in patients aged 26 and over.

Autolus, a spinout from University College London, said it will launch CD19-targeting Aucatzyl "immediately" in England and Wales and also seek approval for the cell therapy from the Scottish Medicines Consortium (SMC).

According to NICE, the final draft guidance means that around 150 people with this form of ALL, who currently have limited treatment options, will likely be eligible for treatment with Aucatzyl over the next three years.

Describing it as a "next-generation" CAR-T, the health technology assessment (HTA) agency said that Aucatzyl has the potential to increase people's likelihood of going into remission and has lower toxicity than other treatments.

Those include two rival CAR-Ts – Novartis' Kymriah (tisagenlecleucel) and Gilead/Kite's Tecartus (brexucabtagene autoleucel) – as well as off-the-shelf alternatives like Amgen's Blincyto (blinatumomab), Pfizer's Besponsa (inotuzumab ozogamicin), and Incyte's Iclusig (ponatinib).

The decision is based on the results of the phase 2 FELIX trial, in which 77% of patients treated with Autolus' CAR-T therapy went into remission. According to NICE's guidance, that makes it roughly equivalent in efficacy to Kymriah, which is available for patients aged 25 and under. The FELIX trial enrolled relatively few patients in that younger age group.

The list price for Aucatzyl, which was approved by the UK medicines regulator in April, is £372,000 per infusion, but will be offered to the NHS at a confidential discount. The is also available in the US for patients aged 18 and over and made sales of around $21 million in the third quarter of this year.

"NHS clinical centres and UK patients participated in the development of Aucatzyl, and we are looking forward to supporting patients and physicians in England and Wales now with the commercial product," said Autolus' chief executive, Dr Christian Itin.

The recommendation has been welcomed by Fiona Hazell, CEO of patient advocacy group Leukaemia UK, who said it "represents an important development in the availability of treatment options for people affected by this type of leukaemia."

NICE's appraisal process for Aucatzyl included patient testimony from Leukaemia UK, as well as the Anthony Nolan organisation, to ensure patients' voices were taken into account.

B-cell ALL is a rare blood cancer, affecting fewer than 5 in 10,000 people in the UK.