Inmune Bio plans to seek approval of RDEB treatment in UK, US, and EU in 2026
Trial of CORDStrom stem cell therapy showed it reduced disease activity
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Inmune Bio plans to submit applications this year seeking regulatory approval in the U.K., the U.S., and the European Union of CORDStrom, its experimental stem cell therapy for recessive dystrophic epidermolysis bullosa (RDEB).
Inmune announced in a press release that it has formally submitted a pre-submission package for CORDStrom to the U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA). A pre-submission package is essentially a request for feedback from the MHRA, intended to streamline an eventual application seeking approval.
Inmune said it expects to get the MHRA’s feedback this summer, after which it plans to formally submit an application seeking approval of CORDStrom in the U.K. The company said it hopes to also submit applications in the U.S. and EU in the fourth quarter of this year, pending feedback from relevant regulatory agencies and manufacturing readiness.
“We are committed to delivering the first systemic therapy for RDEB patients and families, starting in the UK, and look forward to feedback from the MHRA in the hope of accelerating the regulatory and commercial pathway to approval,” said Mark Lowdell, PhD, co-founder and chief scientific officer at Inmune.
Inmune plans to seek approval of CORDStrom based primarily on data from a clinical trial called MissionEB (ISRCTN14409785), which tested the therapy against a placebo in 30 children and adolescents with RDEB. Results announced last year showed that the therapy significantly reduced disease activity, with patients reporting less pain and itching after treatment.
“The Mission EB trial demonstrated improvement in itch, pain, skin integrity and quality of life, with excellent tolerability and no treatment-related serious adverse events,” Lowdell said.
CORDStrom uses stem cells derived from human umbilical cords
RDEB is a rare genetic disorder marked by fragile skin that’s prone to blisters and wounds. Patients often experience itching and pain; the disease is also associated with digestive problems and an increased risk of skin cancer. The only RDEB treatments currently available are applied to the skin and can help with wound healing, but do little to address other manifestations of the disease.
CORDStrom uses stem cells derived from human umbilical cords. These cells, called mesenchymal stem cells, can grow into other cell types and help promote tissue healing. Inmune said it has completed three successful runs of manufacturing a commercial-grade product at scale.
“Meeting the deadline for our pre-submission for CORDStrom marks a critical milestone, building upon the positive safety and efficacy data demonstrated in the MissionEB clinical trial and the successful transition into our commercial manufacturing facility,” Lowdell said.
The U.S. Food and Drug Administration (FDA) has previously granted CORDStrom rare pediatric disease designation and orphan drug status, both of which are designed to incentivize the development of treatments for rare diseases. The rare pediatric disease designation specifically offers drug developers a voucher that can be used to speed review of an experimental treatment. A large spending package recently passed by Congress and signed into law by President Donald Trump earlier this month extended that FDA program through September 2029.
“We welcome Congress’s bipartisan action to extend the rare pediatric disease [priority review voucher] program,” said David Moss, CEO of Inmune. “This reauthorization removes a major uncertainty and strengthens the incentive landscape for advancing innovative treatments for children with devastating rare conditions. With our planned [submission of an application seeking FDA approval of CORDStrom] targeted for later this year, we are well-positioned to potentially benefit from this important incentive as we work to bring CORDStrom to RDEB patients who urgently need new options.”
