Promethera HepaStem is the company's therapy product to treat serions metabolic liver disorders. An European phase IIII clinical trial is ongoing to treat Crigler-Najjar and Urea Cycle Disorders in a pediatric setting. As of today, 20 patients have been treated with HepaStem. Cnrrently, Promethera is preparing its next clinical phases in US and Europe. Th.e challenge is to provide a drug product easy-to-reconstitute at clinical sites. In order to guarantee a flexible, highly quali tative, and economically sustainable supply chain during commercialization, Promethera has developed a ready-to-use off-the-shelf product for direct reconstitution at the hospitals. The product preparation is simply carried out like conventional reconstitution of fi·eeze dried sterile medicinal products (e.g. vaccine). The challenge was to find an appropriate final container (i) compliant to liquid nitrogen storage, (ii) allowing automated in-line filling and (iii) reconstitt1tion without changing the product quality (safety/identity/purity/potency). The Aseptic Technologies closed vials perfectly fit within this concept. This approach does not only simplify the preparation procedure and operation time, it also reduced the storage footprint and improved the product's quality in terms of viability, yield, content uniformity and batch-to-batch consistency. Shelf-life, the key bottleneck of most cell therapeutic products, is substantially less critical. The idea for the upcoming clinical phases is to provide an ali-in-one kit, including the cryopreserved cell tl1erapeutic product HepaStem and all material needed for reconstitution (syringe/adaptor/uisinfectantlprefilled vial with reconsti tution media). In conclusion, Promethera has developed a ready-to-use product, allowing worldwide availability and easy reconstitution at the bedside. This technology guarantees a sustainable supply chain during commercialization and offers opportunities in the field of cell-based products with limited shelf-life.
Snykers, S., P. Willemsen, et al.