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Stem Cell Treatment for Crohn’s Gains Positive EMA Opinion
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Cx601, an investigational allogeneic stem cell therapy for complex perianal fistulas in Crohn’s disease, has received a positive opinion recommending marketing authorization from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), and from the Committee for Advanced Therapies (CAT), according to a press release.
“Following today’s news, physicians and surgeons in Europe can look forward to offering these Crohn’s disease patients a novel and minimally invasive alternative treatment option in the future, which in clinical trials achieved higher combined remission and lower relapse rates than the current standard of care,” Julian Panés, MD, head of the gastroenterology department at the Hospital Clinic of Barcelona in Spain, and president of the European Crohn’s and Colitis Organisation (ECCO), said in the press release. “Perianal fistulas are estimated to affect up to 28% of patients in the first two decades after Crohn’s disease diagnosis and Cx601 offers new hope for those suffering from this severe and debilitating condition.”
Cx601 (darvadstrocel; Takeda/TiGenix) is a suspension of allogeneic (donor derived) expanded adipose-derived stem cells (eASCs) administered locally by intra-lesional injection for the treatment of adult Crohn’s patients with complex perianal fistulas for whom at least one conventional or biologic therapy has been inadequate. The developer, TiGenix, signed a licensing deal with Takeda in July 2016, which granted Takeda exclusive development and commercial rights outside of the U.S. The European Commission granted orphan drug status to Cx601 in 2009, as did the FDA in 2017.
The positive CHMP opinion is supported by results from the phase 3 ADMIRE-CD pivotal trial. As Healio Gastroenterology and Liver Disease previously reported, this was a double-blind randomized controlled trial that demonstrated the compound’s safety and efficacy.
Twenty-four-week data, now published in The Lancet, showed Cx601 was superior vs. placebo for achieving combined remission, the primary efficacy endpoint, and that treatment-related adverse events and discontinuations due to adverse events were comparable between groups. Follow-up data from 52 weeks also showed the compound maintained long-term remission.
“We believe that this first approval recommendation for an allogeneic stem cell therapy in Europe reflects the maturity of our technology and its potential to offer new approaches for difficult to treat conditions,” María Pascual, MD, vice president of regulatory affairs and corporate quality at TiGenix, said in the press release. “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, to fulfill our aim of allowing patients to benefit from the full potential of Cx601 across multiple geographies and diseases.”
The European Commission will now consider the positive opinion, and its decision is expected in the coming months, according to the press release. Marketing authorization would allow Cx601 to be marketed in all 28 EU states, Norway, Iceland and Lichtenstein, and prompt a 15 million-euro milestone payment from Takeda to TiGenix.
“Today’s positive CHMP opinion is a crucial step to bringing a new treatment option to patients with complex perianal fistulas in Crohn’s disease,” Asit Parikh, MD, head of Takeda’s Gastroenterology Therapeutic Area Unit, said in the press release.
“Complex perianal fistulas are considered one of the most disabling complications of Crohn’s disease and can cause intense pain and swelling, infection and incontinence,” and there is currently no effective treatment, according to the press release.
Disclosures: Parikh is employed by Takeda. Pascual is employed by TiGenix. Panés reports financial relationships with Takeda and TiGenix.