Takeda and TiGenix have announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), in conjunction with the Committee for Advanced Therapies (CAT), has adopted a positive opinion recommending a marketing authorisation (MA) for investigational compound Cx601 (darvadstrocel).
Cx601 is expected to be indicated for the treatment of complex perianal fistulas in adult patients with non-active/mildly active luminal Crohn’s disease, when fistulas have shown an inadequate response to at least one conventional or biologic therapy.
The recommendation marks the first allogeneic stem cell therapy to receive a positive CHMP opinion in Europe.
Cx601 was assessed by the CAT, the EMA’s specialised scientific committee for Advanced Therapy Medicinal Products (ATMP), such as gene or cell therapies.
The positive CHMP opinion was based on results from TiGenix’s Phase III ADMIRE-CD pivotal trial. The ADMIRE-CD trial is a randomised, double-blind, controlled, Phase III trial designed to investigate the efficacy and safety of investigational compound Cx601.
24-week results were published in The Lancet and showed that Cx601 achieved statistically significant superiority versus the control group in the primary efficacy endpoint of combined remission. In addition, the rates and types of treatment related adverse events (non-serious and serious) and number of discontinuations due to adverse events were comparable between Cx601 and control arms.
Further follow-up data indicated that Cx601 maintained long-term remission of treatment refractory complex perianal fistulas in patients with Crohn’s disease over 52 weeks.
The opinion will now be referred to the European Commission with a decision anticipated in the coming months. An MA will allow Cx601 to be marketed in all 28 member states of the EU, plus Norway, Iceland and Lichtenstein.
Receipt of the MA will trigger a milestone payment from Takeda to TiGenix of €15 million. The companies have been working closely together to advance preparations for commercialisation, with a potential start of the commercial launch by Takeda anticipated after MA is transferred from TiGenix to Takeda.
Dr. Asit Parikh, Head of Takeda’s Gastroenterology Therapeutic Area Unit, said: “We would like to thank the scientific community and patients involved in the ADMIRE-CD trial for their support in helping us reach this important milestone.”
Dr. María Pascual, VP Regulatory Affairs and Corporate Quality at TiGenix, said: “We have worked closely with the EMA and provided a robust data package from a well-designed clinical trial with challenging endpoints. In parallel, we will continue working hard to obtain regulatory approval in the U.S. and to develop Cx601 for additional indications.”