The National Institute for Health and Care Excellence has announced it will not approve funding for Alofisel, Europe’s first allogeneic stem cell therapy to gain approval for use across the European Union.
Alofisel, a stem cell therapy product developed by TiGenix and licensed to Takeda, was previously approved by the EU in March for the treatment of complex perianal fistulas in adult patients with nonactive/mildly active luminal Crohn's disease, when fistulas have demonstrated an inadequate response to at least one conventional or biologic therapy. Previously, patients in clinical trials who received the treatment showed a 44% higher probability of achieving combined remission compared to the control, with follow-up data at 52 weeks and 104 weeks post-treatment confirmed sustained efficacy and safety.
Draft guidelines from NICE have stated that Alofisel has only demonstrated a modest improvement in the proportion of patients with complex perianal fistulas achieving complete remission compared with placebo in one clinical trial. According to NICE “Reliable follow-up results are only available for up to one year, so it is unclear how long the treatment benefit will last,”, and cost-effectiveness estimates are “highly uncertain” as a result of this and so the committee was unable to reach a plausible cost-effectiveness estimate.
A statement from Takeda says they are "fully committed to collaborating with the IBD community, NICE and NHS England to identify any relevant data which may help to answer the questions raised in NICE’s draft guidance and allow them to make a positive recommendation".