According to a source article on Business Wire that can be found here, the Committee for Orphan Medicinal Products (part of the European Medicines Agency) has recommended that the experimental drug OMS721 be awarded Orphan Drug status for use in hematopoietic stem cell transplantation (HSCT). Omeros Corporation now expects the European Commission to adopt this opinion in August.
About Hematopoietic Stem Cell Transplantation (HSCT)
HSCT, also called blood and marrow transplantation, is used as a treatment for a range of conditions. There are two main forms of HSCT: autologous (when the cells are taken from your own body), and allogeneic (when the cells come from a donor).
The transplant may be used to replace damaged blood cells with healthy ones. It is sometimes carried out after intensive cancer treatments, or in people with other conditions that have caused damage or destruction to their blood cells.
About Orphan Drug Designation
Orphan Drug status is granted by the European Commission for treatments for serious conditions that affect less than five out of every 10,000 people within the European Union.
The designation is designed to support the development of medicines that would significantly benefit patients living with rare diseases, which according to the EMA are estimated to affect 30 million people in Europe. Medicines granted Orphan Drug status are supported through incentives such as protocol assistance (certain scientific advice), a longer period of marketing exclusivity if the product is approved, and, in some cases, reduced fees.
OMS721 is a human monoclonal (one type) antibody, which targets MASP-2, an enzyme involved in the lectin pathway, which is involved in the complement system. This system is important in the immune response, including inflammation responses caused by factors such as tissue damage and some infections.
OMS721 has already received Orphan Drug designation from the US FDA for the prevention of complement-mediated TMA. It has also been awarded Breakthrough Therapy designation for the treatment of stem cell transplant patients with persistent TMA, even after modification of immunosuppressive therapy.
Research into OMS721
Currently, there are three Phase III clinical programs that are investigating OMS721 in relation to HSCT-associated thrombotic microangiopathy (TMA) (typically causing damage to small blood vessels), immunoglobulin A (igA) nephropathy (often linked to kidney damage), and atypical haemolytic uremic syndrome (aHUS) (can cause blood clots to form in the kidneys). In addition, two Phase II clinical studies are being carried out. One is investigating the drug in participants who have igA nephropathy and lupus nephritis, and the second involves patients diagnosed with HSCT-TMA and aHUS.