New guidance expands NHS access to a TTR-silencing therapy for cardiomyopathy and a next-generation CAR-T for relapsed adult B-ALL
The National Institute for Health and Care Excellence (NICE) has delivered two high-impact decisions that shape the UK’s treatment landscape heading into 2026. In final draft guidance, NICE recommended vutrisiran as an option for transthyretin amyloidosis with cardiomyopathy (ATTR-CM), and separately approved Autolus’ Aucatzyl (obe-cel), a next-generation CAR-T therapy for adults with relapsed or refractory B-cell acute lymphoblastic leukaemia (B-ALL). While the two conditions sit on opposite ends of the disease spectrum, cardiac amyloidosis and aggressive blood cancer the decisions reflect a consistent pattern from NICE: sustained support for innovative, high-value therapies that address severe unmet need.
What You Need To Know
- NICE recommends vutrisiran for routine NHS use in adult ATTR-CM, citing comparable clinical outcomes and cost to tafamidis.
- HELIOS-B data show reduced mortality and cardiovascular events versus placebo, supporting its long-acting, quarterly dosing model.
- NICE approves Autolus’ Aucatzyl CAR-T for adults with relapsed or refractory B-ALL, with 77% remission in clinical trials.
- Both decisions expand access to disease-modifying and potentially curative therapies for high-need cardiac and oncology populations.
Vutrisiran lands routine NHS access for ATTR-CM
NICE’s final draft guidance confirms that vutrisiran can be used, within its marketing authorisation, to treat adults with wild-type or hereditary ATTR-CM when provided under its commercial access agreement . The therapy, a subcutaneous transthyretin (TTR) silencer administered once every three months, becomes a routine NHS option within 90 days of publication.
The committee concluded that vutrisiran delivers comparable long-term outcomes to tafamidis, the current standard of care, offering similar or lower overall cost to the system. Data from the HELIOS-B trial showed significant reductions in all-cause mortality and recurrent cardiovascular events compared with placebo, including in patients not receiving background tafamidis at baseline . NICE judged the comparative evidence between vutrisiran and tafamidis to have uncertainty but ultimately sided with the view that both therapies offer equivalent clinical effectiveness at current pricing.
NICE’s accompanying news summary emphasises the therapy’s long-acting, home-administered profile. Vutrisiran is the first and only disease-modifying ATTR-CM treatment with quarterly dosing, a notable advantage for a patient population that is often elderly, frail, or geographically distant from specialist centres. Around 1,500 patients in England and Wales could be eligible, according to the published report .
Clinicians quoted in the NICE news release underscored the significance of targeting the “root cause” of ATTR-CM by halting production of misfolded TTR proteins. They also highlighted the burden of delayed diagnosis, where patients frequently present after months or years of symptoms, making access to a long-acting, disease-modifying therapy even more critical.
Aucatzyl CAR-T approved for relapsed or refractory B-ALL
In a separate decision, NICE recommended Autolus Therapeutics’ Aucatzyl (obecabtagene autoleucel) for adults aged 26 and over with relapsed or refractory B-cell precursor acute lymphoblastic leukaemia. The therapy will be commissioned routinely across NHS England and Wales, delivering a long-awaited option for a population with limited alternatives and historically poor outcomes.
NICE’s announcement reports that over 150 patients are expected to benefit in the next three years . Aucatzyl demonstrated a 77% remission rate in a nearly 100-patient clinical trial cohort, with evidence suggesting improved survival compared with currently available salvage immunotherapies. The committee applied greater weighting to the treatment’s benefits due to the severity of late-line B-ALL.
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Autolus’ press release expands on these results, noting that Aucatzyl uses a proprietary CD19 CAR design intended to reduce toxicity while maintaining robust anti-leukaemic activity. The therapy earned conditional MHRA approval in April 2025 based on the FELIX trial and is now positioned as a next-generation CAR-T option specifically for adults, complementing tisagenlecleucel, which is approved in younger populations .
Patient organisations and UK life sciences leaders welcomed the decision, calling it a landmark for UK-developed cell therapy. Several cancer alliances highlighted how the NIHR-supported clinical ecosystem helped accelerate evaluation and delivery, enabling patients to gain access shortly after approval.
For media inquiries or to share perspectives on access and evidence policy, email editor@synopulse.com.
