TiGenix and Takeda announced that the European Commission (EC) has approved Alofisel (darvadstrocel) for the treatment of complex perianal fistulas in adult patients with nonactive/mildly active luminal Crohn’s disease, marking the first allogeneic stem cell therapy to receive central marketing authorization approval in Europe.
Crohn’s disease is a chronic condition that causes intestinal inflammation. A complication of the disease is the development of perianal fistulas that occur when an abnormal passageway develops between the perianal space and the outside skin surface. These can lead to incontinence (a lack of control over the opening of the bowels) and sepsis (blood infection). Complex fistulas are known to be more treatment resistant than simple fistulas. There is currently no cure for Crohn’s disease, so current treatment options for complex fistulas in patients with Crohn’s disease include the use of antibiotics, immunosuppressants, anti TNFs and surgery. However, these treatment options are marked by poor long term efficacy and considerable safety issues.
Alofisel contains expanded adipose stem cells that impair proliferation of lymphocytes and reduce the release of pro-inflammatory cytokines at inflammation sites. This immunoregulatory activity stimulated by local administration of Alofisel reduces inflammation and may allow the tissues around the fistula tract to heal.
The European approval follows a positive opinion by the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP), in conjunction with the Committee for Advanced Therapies (CAT), in December 2017. The recommendation was based on results from TiGenix’s Phase III ADMIRE-CD pivotal trial, which showed that Alofisel achieved statistically significant superiority versus the control group in the primary efficacy endpoint of combined remission at 24 weeks, as well as further follow-up data that indicated Alofisel maintained long-term remission of treatment refractory complex perianal fistulas in patients with Crohn’s disease over 52 weeks.
“This approval of Alofisel reflects our deep understanding and recognized leadership in the development of allogeneic stem cells and our firm commitment to developing innovative therapies for medical needs,” said Dr. María Pascual, VP Regulatory Affairs and Corporate Quality at TiGenix. “We are pleased to offer the medical community an important new treatment option for patients with Crohn’s disease who do not respond to currently available therapies.”
“Today’s marketing authorization, the first for an allogeneic stem cell therapy, represents a positive advancement in the treatment of patients with complex perianal fistulas in Crohn’s disease,” said Dr. Asit Parikh, Head of Takeda’s Gastroenterology Therapeutic Area Unit. “We look forward to bringing this much needed treatment option to patients across Europe in the coming months.”
The approval of this allogeneic stem cell therapy highlights the ability of regenerative medicine to offer new treatment options to patients who do not respond to existing therapies.