INmune Bio wins MHRA PIP approval for Ebstrocel in RDEB
INmune Bio has secured approval of the Paediatric Investigation Plan (PIP) for Ebstrocel from the UK Medicines and Healthcare products Regulatory Agency (MHRA), a prerequisite for any Marketing Authorisation Application (MAA) in the United Kingdom. The Florida-headquartered, NASDAQ-listed company said the approval came in under three months, against an anticipated ten-month review cycle, and keeps its planned 2026 MAA filing firmly on schedule.
Ebstrocel is derived from the company's CORDStrom platform, which uses pooled, allogeneic, human umbilical cord-derived mesenchymal stromal cells (hucMSCs) formulated as an off-the-shelf injectable. It is being developed for Recessive Dystrophic Epidermolysis Bullosa (RDEB), a rare and severe genetic skin fragility disorder in which the absence of functional type VII collagen leads to chronic blistering, wounds, and systemic complications. INmune Bio describes RDEB as a systemic disease rather than a pure skin condition, positioning Ebstrocel as a potential first systemic therapy targeting its underlying disease burden.
Regulatory path
Mark Lowdell, Chief Scientific Officer, said the MHRA's accelerated turnaround "represents a clear endorsement of the clinical data package" and confirmed that minor protocol adjustments to the Phase 3 open-label trial have already been incorporated. The approval also carries broader significance: the MHRA has now reviewed and provided written alignment across the chemistry, manufacturing and controls (CMC), non-clinical and clinical strategy components needed for the full MAA.
INmune Bio is pursuing a twin-track strategy in the UK and EU. It plans to file for Conditional Marketing Authorisation in 2026, aiming to enable early market access, while a confirmatory open-label registration trial in 2027 would underpin full authorisation. A parallel US Biologics Licence Application (BLA) filing is planned for early 2027, with the company having previously secured FDA Accelerated Approval designation for the programme.
Chief executive David Moss described the milestone as "a significant regulatory de-risking event" and said accepted meeting minutes from a face-to-face Scientific Advice session with the MHRA provide a clear roadmap through to conditional approval, covering the evidence package, manufacturing transition, and confirmatory trial design.
Market context
The rare skin disease space has attracted growing attention from cell therapy and biologics developers, driven by orphan drug incentives on both sides of the Atlantic and by the severity of conditions such as RDEB, which carries a heavy burden of chronic pain, wound care costs, and shortened life expectancy. The UK patient population for RDEB is estimated at approximately 300 children, part of a broader epidermolysis bullosa community of at least 5,000 individuals supported by the charity DEBRA UK.
The competitive landscape for RDEB remains limited: Krystal Biotech's B-VEC, a gene therapy topical gel approved by the FDA in 2023, addresses wound healing but does not target systemic disease. A systemic cell therapy with an off-the-shelf manufacturing profile would occupy a distinct clinical niche. CORDStrom's pooled donor approach is designed to reduce the batch-to-batch variability that has historically constrained mesenchymal stromal cell programmes, though regulators and payers will want to see the confirmatory trial data before drawing conclusions on durability.
For INmune Bio, the MHRA milestone follows its earlier End-of-Phase 2 alignment meeting with the FDA on its separate XPro1595 neuroinflammation programme, suggesting the company is managing two late-stage regulatory tracks simultaneously. Near-term investor focus will shift to the timing of the formal MAA submission and the commercial manufacturing readiness steps the company says are its current priority.