Marketing authorization of darvadstrocel for treatment of refractory complex perianal fistulas in patients with Crohn's disease has been recommended by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).
Darvadstrocel is a suspension of allogeneic expanded adipose-derived stem cells. It is the first allogeneic stem cell therapy to receive a positive Committee for Medicinal Products for Human Use opinion in Europe, the company notes.
Dr Julian Panés, head of gastroenterology at the Hospital Clinic of Barcelona in Spain and president of the European Crohn's and Colitis Organization said perianal fistulas are estimated to affect up to 28% of patients in the first two decades after Crohn's disease diagnosis.
The darvadstrocel provides new hope for the individuals who are suffering from the severe and debilitating condition called perianal fistulas, he added. In clinical trials, darvadstrocel (formerly Cx601) achieved higher combined remission and lower relapse rates than the current standard of care.
In phase 3 ADMIRE-CD trial, the benefits of darvadstrocel were demonstrated. The trial involved 212 patients with Crohn's disease and complex perianal fistulas whose condition did not respond to one or more therapies, including antibiotics, immunosuppressants, and tumour necrosis factor–alpha inhibitors.
The researchers compared about half of the patients treated with darvadstrocel were in remission, to about a third of patients treated with placebo after 6 months of treatment. With darvadstrocel, long-term remission of complex perianal fistulas was maintained in patients with Crohn's disease for longer than 52 weeks.
Although there is a moderate difference between the treatment groups, the effect is considered to be clinically meaningful when other treatment options for fistulas have failed, the EMA said in a statement.
The most commonly observed side effects include anal abscess and fistula, as well as procedural pain and proctalgia. The positive CHMP opinion will now be sent to the European Commission. A decision is anticipated in the coming months, the company said.
In conclusion, the phase 3 ADMIRE-CD trial showed the benefits of darvadstrocel. With darvadstrocel, long-term remission of complex perianal fistulas was maintained in patients with Crohn's disease for longer than 52 weeks, the study reported.