INmune Bio has secured MHRA approval of its Pediatric Investigation Plan for Ebstrocel™ in recessive dystrophic epidermolysis bullosa, marking the first regulatory step toward a UK marketing application in 2026 and supporting a dual‑track strategy for accelerated approval in the US.
Written By: Fariha Sameen, PharmD
Reviewed By: Pharmacally Editorial Team
INmune Bio has received approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) for the Pediatric Investigation Plan (PIP) for Ebstrocel™ in the treatment of recessive dystrophic epidermolysis bullosa (RDEB). The approval represents the first mandatory regulatory step before submitting a Marketing Authorization Application (MAA) in the United Kingdom and keeps the company on track for a 2026 filing.
The company also plans to pursue accelerated approval in the United States, with a regulatory submission expected in early 2027.
Ebstrocel Uses Umbilical Cord-Derived Cells to Treat Systemic Disease
Ebstrocel is the lead investigational therapy developed using INmune Bio’s CORDStrom™ platform. The therapy consists of pooled, allogeneic human umbilical cord-derived mesenchymal stromal cells (hucMSCs) manufactured as an off-the-shelf cell therapy.
Rather than targeting only skin lesions, Ebstrocel addresses the systemic inflammation associated with RDEB. The therapy is intended to improve chronic wounds, severe itching, persistent pain, and other complications that significantly reduce quality of life.
RDEB is a rare inherited disorder caused by mutations affecting skin integrity. Patients experience fragile skin, recurrent blistering, chronic wounds, scarring, and a markedly increased risk of aggressive squamous cell carcinoma. Approximately 300 children in the UK are affected by RDEB, while more than 5,000 people live with various forms of epidermolysis bullosa.
MHRA Review Supports Regulatory Strategy
The company expected the PIP review to take around 10 months but requested an accelerated assessment because of the severe unmet medical need. The MHRA completed its review in less than three months.
According to INmune Bio, the agency raised no concerns regarding the clinical evidence supporting pediatric use. The regulator recommended only minor modifications to the Phase 3 open-label protocol, which have already been incorporated into the development program.
The approved PIP establishes the scientific rationale for treating children with Ebstrocel and supports the company’s planned submission for Conditional Marketing Authorization later this year. A confirmatory open-label registration study is scheduled to begin in 2027 to satisfy post-marketing requirements for full approval.
Regulatory Alignment Reduces Development Risk
Chief Scientific Officer Dr. Mark Lowdell said the rapid review validates the clinical package and allows the company to continue its dual-track strategy of pursuing conditional approval while generating confirmatory evidence.
Chief Executive Officer David Moss added that recent scientific advice from the MHRA has aligned the company on manufacturing, non-clinical, clinical, and chemistry, manufacturing and controls (CMC) requirements. He noted that this agreement substantially reduces regulatory uncertainty ahead of the planned MAA submission and provides a clear roadmap toward commercialization.
Path Forward
INmune Bio is completing commercial manufacturing activities and final regulatory preparations before submitting its UK Marketing Authorization Application later in 2026. If approved, Ebstrocel could become one of the first systemic cell therapies available for patients with RDEB, addressing the underlying disease burden beyond wound management while expanding treatment options for this rare and debilitating condition.
Reference
INmune Bio Inc. Achieves MHRA Approval of PIP for CORDStrom™
About the Writer
Fariha Sameen, PharmD (LinkedIn), is a clinical pharmacy professional with hands-on experience in patient counselling, medication review, therapeutic monitoring, and clinical documentation across multiple departments. She has experience identifying and assessing drug-related problems and supporting medication safety practices. Her interests include pharmacovigilance, ADR reporting, clinical research, and medical writing focused on clear, evidence-based communication.