Cost regulators for the NHS in England and Wales have not approved funding for TiGenix and Takeda’s Alofisel - the first allogeneic stem cell therapy to be approved for use across the European Union – for use in Crohn’s patients.
The therapy (previously referred to as Cx601) was approved in March to treat complex perianal fistulas in adult patients with nonactive/mildly active luminal Crohn's disease, when fistulas have shown an inadequate response to at least one conventional or biologic therapy.
Perianal fistulas, a common complication of Crohn’s disease, occur when an abnormal passageway develops between the rectum and the outside of the body, potentially leading to incontinence and sepsis. Complex fistulas, which are rare, are more treatment resistant than simple fistulas.
Alofisel (darvadstrocel) is a local administration of allogeneic (donor derived) expanded adipose-derived stem cells (eASCs).
In clinical trials underpinning the drug’s approval, patients receiving the treatment showing a 44 percent greater probability of achieving combined remission compared to placebo, while a follow-up analysis (at 52 weeks and 104 weeks post-treatment) confirmed sustained efficacy and safety, according to the firms.
However, in draft guidelines, the National Institute for Health and Care Excellence said Alofisel showed only a modest improvement in the proportion of people with complex perianal fistulas achieving complete remission compared with placebo in one clinical trial.
“Reliable follow-up results are only available for up to one year, so it is unclear how long the treatment benefit will last,” according to the guidelines.
Because of this, cost-effectiveness estimates are “highly uncertain” and the committee was unable to conclude on the most plausible cost-effectiveness estimate, NICE said.
Takeda said it is "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community, NICE and NHS England to identify any relevant data which may help to answer the questions raised in NICE’s draft guidance and allow them to make a positive recommendation".