Alofisel®（darvadstrocel）在日本获批用于治疗克罗恩病患者的复杂肛周瘘管

On September 27, 2021, Takeda Pharmaceutical Co., Ltd. announced that it has received approval from the Ministry of Health, Labor and Welfare of Japan to produce and sell Alofisel (darvadstrocel) for the treatment of complex perianal fistulas in patients with inactive or mildly active cast Crohn's disease (CD).

Crohn's disease is a chronic inflammatory disease of the digestive system that affects an estimated 70,700 people in Japan. People with Crohn's disease may develop complex perianal fistulas, which can cause severe pain, bleeding, swelling, and infection. Despite medical and surgical advances, complex perianal fistulas in patients with Crohn's disease remain a challenging condition for clinicians. In a survey commissioned by Takeda and commissioned by the European Federation of Crohn's and Ulcerative Colitis Associations (EFCCA), the quality of life of Crohn's disease patients with perianal fistulas was negatively affected in many areas, including exercise capacity, work, employment, romantic relationships and sex life. Patients also report that they feel more unhygienic, uncomfortable, and feel guilty about it than people with Crohn's disease who do not have perianal fistulas.

The Darvadstrocel-3002 study is a Phase 3, multicenter, open-label, uncontrolled study designed to investigate the efficacy and safety of Alofisel in the treatment of complex perianal fistulas in 22 Japanese adult patients with inactive/mildly active intestinal CD.

ADMIRE-CD was a randomized, double-blind, controlled Phase 3 trial investigating the efficacy and safety of Alofisel in the treatment of complex perianal fistulas in 212 adult patients with inactive/mildly active intestinal CD. At 24 weeks of follow-up, significantly more patients in the Alofisel group achieved the primary endpoint of combined response compared with the control group, which was maintained through 52 weeks. Alofisel treatment was well tolerated through 52 weeks, with a safety profile similar to that of the control group.

Alofisel is the first expanded human allogeneic adipose-derived mesenchymal stem cell therapy approved in Japan. By exhibiting immunomodulatory and local anti-inflammatory effects at the site of inflammation, it provides a new treatment option for complex perianal fistulas in patients with inactive or mildly active intestinal Crohn's disease.

References:

https://www.takeda.com/newsroom/newsreleases/2021/takeda-receives-approval-to-manufacture-and-market-alofisel-darvadstrocel-in-japan-for-treatment-of-complex-perianal-fistulas-in-patients-with-non-active-or-mildly-active-luminal-crohns-disease/