Takeda bags Japanese approval for Crohn's cell therapy Alofisel

Takeda has chalked up another milestone for its Alofisel cell therapy for a complication of Crohn's disease, becoming the first allogeneic stem cell therapy to be approved in Japan.

Alofisel (darvadstrocel) has been cleared by the Ministry of Health, Labour and Welfare (MHLW) for the treatment of complex perianal fistulas in patients with non-active or mildly active luminal Crohn's disease who have not responded to conventional therapies.

Some patients with Crohn's disease can develop fistula, which is a channel that connects the anal canal or rectum to the surface of the skin near the anus. They can cause intense pain, bleeding, swelling, infection, and anal discharge.

Around a third of these can require surgery because drug treatment with antibiotics, anti-inflammatory biologics like TNF inhibitors or immunosuppressants such as methotrexate or ciclosporin don't encourage them to heal.

Alofisel – previously known as Cx601 – offers another treatment option before surgical intervention. When injected close to the perianal fistulas, the cell therapy reduces inflammation, increasing the likelihood of the fistulas healing.

It consists of stem cells which are taken from the fat tissue of a healthy adult donor and then grown in a laboratory. It is a made to order therapy that must be administered within 72 hours of manufacture.

In a phase 3 trial, 52% of patients treated with Alofisel were in remission after 26 weeks, compared to 36% of a control group, rising to 56% and 39% respectively after a year.

Alofisel is already approved in the EU, UK, Switzerland and Israel but suffered a setback when cost-effectiveness agency NICE declined to recommend it for NHS use in England and Wales, citing modest benefit in clinical trials, even though Takeda offered to cover the cost of treatment if it was not successful.

Its counterpart in Scotland – the SMC – subsequently also turned down the therapy. Sales have started to pick up but remain low, rising from just 11 million yen in the three months to June 30 last year to 388 million yen (around $3.5 million) in the same period of 2021.

Alofisel was initially developed by Belgian biopharma TiGenix, was acquired by Takeda for around $600 million in 2018.