dm+d

Unassigned

New Medicines

Recessive dystrophic epidermolysis bullosa (RDEB)

Information

New molecular entity
RHEACELL
RHEACELL

Development and Regulatory status

Phase II Clinical Trials
Phase II Clinical Trials
Phase II Clinical Trials
Yes
Yes
May 22A PIII trial is in set up [4].
May 22APZ2 has orphan drug status in the EU and US [4].

Category

An allogeneic skin-derived ATP-binding cassette sub-family B member 5 (ABCB5) positive mesenchymal stem cell therapy. ABCB5 expressing cells are totipotent in nature and are generally expressed in extra-embryonic tissues. These cells have multiple clinical applications due to their immunomodulatory functions and differential plasticity.
Population studies are complicated by the fact that this is not a uniform disease but a wide number of diseases of varying severity. It is likely that many patients with the more mild forms of EB remain undiagnosed. It is thought to affect 1 in 17,000 births. Around 5,000 people are affected in the UK [1]. RDEB is caused by mutations in the COL7A1 gene, which disrupt the production of type VII collagen and impairs its ability to help connect the epidermis to the dermis [2].
Recessive dystrophic epidermolysis bullosa (RDEB)
Intravenous infusion

Trial or other data

Jul 20PI/IIa study (NCT03529877) completes after enrolling 16participants. Company announces that intravenous APZ 2 significantly reduced disease activity, as indicated by improvements in local wound environment and significant improvements in the overall health of the treated population [2,3].
Jan 19PI/IIa trial to evaluate the efficacy and safety of APZ 2 (allo-APZ2-EB) for the treatment of patients with RDEB starts (allo-APZ2EB-II01; EudraCT2018-001009-98; NCT03529877). The trial will enrol 40 patients, aged up to 55 years, in the US, Austria, France, Germany, Italy and UK (sites King´s College London and Great Ormond Street Hospital). Patients will undergo treatment with the IMP (three repeated intravenous applications) and will be followed up for efficacy for 12 weeks. To assess long-term safety of allo-APZ2-EB one follow-up visit at Month 12 and one follow-up visit at Month 24 post IMP applications is included. Determination of the EB linked symptoms and quality of life will be assessed by using the EBDASI score, the iscorEB, the change in pain and itch perception, and patient´s quality of life in EB. The wound healing process will be documented by photography. Collection of primary outcome data (change in EBDASI score and adverse events) is due to complete Dec 21 [3].