Japan’s Ministry of Health, Labour, and Welfare has just announced its approval of Alofisel, a stem cell therapy created by Takeda. This approval is specifically for patients who are diagnosed with non-active or mild active luminal Crohn’s disease (CD) who are facing perianal fistulas and who have not responded to other therapies.
This approval means that Takeda is able to manufacture and market the new treatment for Crohn’s disease patients in Japan. It is the very first expanded human allogeneic adipose-derived mesenchymal stem cell therapy to be approved in Japan.
Crohn’s disease is an inflammatory condition which impacts the digestive tract. One of the complications which can ensue from this disease is complex perianal fistulas.
These cause pain and bleeding and may cause swelling, discharge, and infection. They can significantly impact a patient’s quality of life. Patients have documented that they can impact their employment, their dating life, their physical activity, and more. They are uncomfortable, and patients often report feeling unhygienic. Unfortunately, they remain very difficult to treat.
This new approval of Alofisel could be life changing for patients who don’t respond to either conventional therapies or biologic therapies.
Alofisel is an anti-inflammatory and immunomodulatory therapy. It works directly at the place of inflammation.
The therapy is a suspension of allogenic expanded adipose-derived stem cells. It was first given Orphan Drug Designation in 2009 by the European Commission. Then, in 2017, it was granted this designation by the FDA. Finally, in 2019 it was given this designation by the Japanese Ministry of Health, Labour, and Welfare.
The therapy is currently approved for complex perianal fistulas in Israel, the European Union, the United Kingdom, and Switzerland.
This therapy is made to order, and it must be administered to patients within the first 72 hours after it is manufactured. Thankfully, Takeda’s manufacturing system allows the treatment to be delivered carefully. The company employs strict temperature regulations.
The Clinical Trials
The approval of this therapy is the result of two different clinical trials: Darvadstrocel-3002 and ADMIRE-CD. The first trial was conducted in Japan and the second was conducted in Israel and Europe.
This Phase 3 trial studied the safety and the efficacy of Alofisel in 22 adult Japanese patients who were diagnosed with complex perianal fistulas.
All patients had mild or non-active Crohn’s disease.
ADMIRE-CD was a Phase 3 randomized trial which studied both the safety and the efficacy of Alofisel. It included 212 patients with complex perianal fistulas. All patients in the trial were diagnosed with Crohn’s disease which was nonactive or mild.
This study demonstrated that 5.15% of those given Alofisel reached remission at 24 weeks (the primary endpoint of the trial). Meanwhile, in the placebo group, only 35.6% reached remission by this time.
Further, this statistically significant difference was maintained until the end of the trial (52 weeks).
This trial also demonstrated that the treatment was well-tolerated and safe for patients.
Click here to learn more about this approval.