Takeda Pharmaceutical Company has received Japan’s Ministry of Health, Labour and Welfare (MHLW) approval for Alofisel (darvadstrocel) to treat certain types of fistulas.
The drug was indicated for the treatment of complex perianal fistulas in patients with non/mildly active luminal Crohn’s disease (CD), who do not respond to existing treatment.
CD is a chronic inflammatory disease that affects the digestive system of people, causing complex perianal fistulas with intense pain, bleeding, swelling, infection, and anal discharge.
Alofisel is a suspension of allogeneic expanded adipose-derived stem cells (eASC) intended for the treatment of complex perianal fistulas in adults with non-active/mild luminal CD.
The drug delivers immunomodulatory and local anti-inflammatory effects at the site of inflammation, and offers a new treatment option for complex perianal fistulas.
It was authorised in the European Union (EU) in March 2018, to treat complex perianal fistulas in adult patients with non-active/mild luminal CD.
In 2019, Alofisel has received the US FDA Regenerative Medicine Advanced Therapy (RMAT) designation for complex perianal fistulas in adults with CD.
Takeda Development Centre Japan general manager Naoyoshi Hirota said: “We are delighted that Alofisel has been approved in Japan for the treatment of complex perianal fistulas in Crohn’s disease patients.
“Complex perianal fistulas are a challenging complication of Crohn’s disease, and the approval of the first expanded human allogeneic adipose-derived mesenchymal stem cell therapy in Japan offers a potential cell-mediated closure option to patients who do not respond to conventional or biologic therapies.”
The regulatory approval is supported by data from two clinical trials, the Darvadstrocel-3002 study conducted in Japan, and the ADMIRE-CD trial in Europe and Israel.
Darvadstrocel-3002 was a Phase 3, multicentre, open-label, uncontrolled study that assessed the efficacy and safety of Alofisel for the treatment of complex perianal fistulas.
ADMIRE-CD was a Phase 3 trial that evaluated the efficacy and safety of Alofisel for the treatment of complex perianal fistulas in patients with non-active/mildly active luminal CD.
In the clinical trials, treatment using Alofisel achieved the primary endpoint of combined remission at a 24-week follow-up and was maintained for 52 weeks.
The treatment was well-tolerated, with a safety profile similar to the control group.
Takeda claimed that Alofisel is the first expanded human allogeneic adipose-derived mesenchymal stem cell therapy to be approved in Japan.
Furthermore, the complex perianal fistulas drug is a made-to-order therapy that needs to be administered within 72 hours of manufacture.
The Japanese drugmaker has created a production and logistics system in Japan for the delivery of the drug across the nation, under strict temperature control.
