Autolus closes on EU okay for leukaemia cell therapy

 Autolus' CAR-T cell therapy for a rare and aggressive form of acute lymphoblastic leukaemia (ALL), Aucatzyl, has been recommended for conditional approval in the EU.

The EMA's human medicines committee, the CHMP, has issued a positive opinion of Aucatzyl (obecabtagene autoleucel) as a treatment for relapsed or refractory B-cell precursor ALL in patients aged 26 and over, according to London, UK-based Autolus. The decision puts it on course for approval in the latter half of this year.

The decision is based on the results of the phase 2 FELIX trial, in which 64% of patients treated with the CD19-targeting CAR-T therapy had a durable response over a median follow-up period of 14 months, including 49% complete responses.

As part of the conditional approval recommendation, Autolus has been asked by the EMA to submit long-term follow-up results from FELIX and to run a non-interventional, real-world evidence (RWE) study based on a patient registry.

Aucatzyl, which was approved in the US last year and in the UK last month, is competing in the marketplace with other CAR-T therapies for B-cell precursor ALL, including Novartis' Kymriah (tisagenlecleucel) and Gilead/Kite's Tecartus (brexucabtagene autoleucel). Sales in the US were $9 million in the first quarter of this year.

The UK company reckons that its CAR-T has the advantage of improved tolerability compared to its rivals. Including relatively low rates of cytokine release syndrome (CRS) and neurotoxicity, which are recognised and potentially serious side effects seen with this type of cell therapy.

It is also developing the CAR-T for follow-up indications, including lupus nephritis, in which a phase 2 trial is due to start later this year, and progressive multiple sclerosis.

Other CHMP decisions

At its May meeting, the CHMP also recommended approval of GSK's BCMA-targeting antibody-drug conjugate (ADC) Blenrep (belantamab mafodotin) as a treatment for relapsed or refractory multiple myeloma, continuing a comeback for the once-withdrawn therapy, and Roche's PI3K inhibitor Itovebi (inavolisib) for HR-positive, HER2-negative advanced breast cancer with PIK3CA mutations.

There were also two positive opinions for new rare disease therapies, namely SpringWorks' Ezmekly (mirdametinib) for neurofibromatosis type 1 – which could soon become a Merck KGaA product if a planned $3.9 billion acquisition goes through – and Recordati's Maapliv (amino acids) for the treatment of genetic disorder maple syrup urine disease (MSUD) in patients from birth.

The committee also recommended approval of Neuraxpharm's Riulvy (tegomil fumarate) for the treatment of adults and children from 13 years of age with relapsing-remitting MS.

However, it turned down applications from a formulation of atropine intended to treat myopia in children from FGK as well as 4SC's Kinselby (resminostat) as a treatment for mycosis fungoides and Sezary syndrome, two cancers of blood cells that affect mainly the skin.

The latter decision comes after the EMA signalled that it would also not be approving the drug for cutaneous T-cell lymphoma (CTCL), its lead indication, putting 4SC's prospects in doubt as Kinselby is its only pipeline product.

https://pharmaphorum.com/news/autolus-closes-eu-okay-leukaemia-cell-therapy