Inmune gearing up for applications seeking approval of RDEB cell therapy
Filings for Ebstrocel start in the UK this year, followed by the US and EU in 2027
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Inmune Bio plans to submit regulatory applications in the U.K., U.S., and European Union seeking approval of Ebstrocel, its experimental stem cell therapy for recessive dystrophic epidermolysis bullosa (RDEB).
The company expects to file its application in the U.K. before the end of this year, followed by planned submissions in the U.S. and EU in early 2027, according to a press release.
“Our focus is on converting the clinical, regulatory and manufacturing progress achieved to date into a defined sequence of registration milestones across the UK, European Union and United States. Each step advances Ebstrocel toward patients,” said David Moss, co-founder and CEO of Inmune.
Inmune has already prepared its manufacturing process for commercial-grade Ebstrocel, and recently engaged with EU regulators to prepare for its application there. As part of that process, Inmune asked EU authorities to grant orphan medicinal product designation for Ebstrocel in RDEB.
Orphan drug designation provides economic incentives to companies developing treatments for rare diseases where, by definition, only a small number of patients might use the treatment. In the EU, the designation offers benefits such as a decade of guaranteed market exclusivity. Inmune’s stem cell platform, named CORDStrom, has already received orphan drug status in the U.S.
Regulatory steps in Europe
Inmune also submitted a scientific advice package to the European Medicines Agency (EMA). This formal step clarifies regulatory requirements ahead of the main application to help streamline the review process.
“These EMA submissions are another execution milestone as we move Ebstrocel from late-stage clinical development toward potential commercialization,” Moss said.
RDEB is a genetic disorder that causes severe skin fragility, leaving patients vulnerable to painful wounds and blisters. Ebstrocel contains stem cells called mesenchymal stromal cells (MSCs) derived from human umbilical cords that are intended to promote wound healing. The therapy is administered as an infusion into the bloodstream.
“RDEB is a systemic disease with substantial unmet need beyond the visible manifestations of skin injury. Advancing Ebstrocel through European regulatory pathways is important for patients,” said Mark Lowdell, PhD, chief scientific officer of Inmune.
The company plans to support its approval applications using data from a clinical trial called MissionEB (ISRCTN14409785), which tested Ebstrocel against a placebo in 34 children with intermediate or severe RDEB.
The trial’s main goal was to determine whether Ebstrocel improved overall disease activity, as measured by the Epidermolysis Bullosa Disease Activity and Scarring Index. Results showed that Ebstrocel treatment did not change overall scores, but it did reduce itch and pain, especially in patients with less severe disease. In interviews, families in the trial viewed the experimental treatment favorably.
If Ebstrocel is approved, Inmune hopes to develop other MSC therapies using its CORDStrom platform.
“Ebstrocel is our first opportunity to demonstrate how a pooled, scalable, off-the-shelf [MSC] platform can progress through late-stage regulatory development,” said Lowdell, the inventor of the CORDStrom platform.
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