Osiris immune drug gets orphan designation

In Europe, orphan drug designation provides market exclusivity for up to 10 years to companies that develop drugs for underserved patient populations. Prochymal has already been granted both orphan drug and fast track status by the FDA, expediting the development of the stem cell treatment.

Prochymal is currently being evaluated in a phase III study for the treatment of acute graft vs. host disease (GVHD). This ongoing phase III study is anticipated to be the final trial before the product is submitted for full approval. Following positive phase II results, Osiris also received clearance to initiate a phase III trial for Prochymal for the treatment of Crohn’s disease.

“We appreciate the recognition from the EMEA and believe that Prochymal has the potential to be the first treatment available for this devastating disease,” said Moya Daniels, director of Prochymal.

GVHD and Crohn’s disease are cell-mediated inflammatory processes that result in high levels of pro-inflammatory chemical signals called cytokines. These cytokines cause the unbalanced activation of certain immune cells that result in tissue damage. Laboratory data indicate that Prochymal is able to down-regulate the production of pro-inflammatory cytokines, including tumor necrosis factor-alpha or TNF-alpha and interferon-gamma.