MorphoSys AG, one of the world's leading biotechnology companies focusing on fully human antibodies, has received the US Food & Drug Administration (FDA) orphan drug designation to MOR208, its humanized Fc engineered monoclonal antibody against CD19, for the treatment of chronic lymphocytic leukaemia (CLL) or small lymphocytic leukaemia (SLL). Additionally, the company has received a positive opinion from the European Medicines Agency (EMA) for an orphan medicinal product application for MOR208 in these indications.
"We are pleased that the FDA and the EMA support our applications for an orphan drug and orphan medicinal product status for MOR208 for the treatment of CLL or SLL. These are important regulatory milestones for the program," commented Dr. Arndt Schottelius, chief development officer of MorphoSys AG. "We will continue to work closely with the FDA and the EMA as we advance MOR208 through clinical development and the associated regulatory processes."
Orphan drug and orphan medicinal product status are granted by the US and European health authorities respectively to promote the development of promising therapeutics for the treatment of rare diseases affecting fewer than 200,000 people in the US annually and no more than 5 in 10,000 people in the European Union. Orphan drug designation includes benefits such as a seven-year period of marketing exclusivity in the United States and 10 years of market exclusivity in the European Union after approval. Other potential advantages come in the form of protocol assistance, the ability to apply for research funding, tax credits for certain research expenses, and fee waivers for the regulatory procedures.
MOR208 is a humanized monoclonal antibody that targets the antigen CD19 for treatment of B cell malignancies and autoimmune diseases. The program is currently in phase 2 clinical development in CLL, acute lymphoblastic B-cell leukaemia and non-Hodgkin's lymphoma. MOR208 has been engineered to possess significantly enhanced antibody-dependent cell-mediated cytotoxicity (ADCC), thus improving a key mechanism for tumour cell killing and offering potential for enhanced efficacy compared to traditional antibodies for the treatment of cancer. The program was in-licensed from Xencor in 2010.