AstraZeneca and Acerta Pharma BV, a company in which AstraZeneca has a majority equity investment, announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) adopted three positive opinions recommending acalabrutinib (ACP-196) for designation as an orphan medicinal product. The three positive opinions are for the treatment of chronic lymphocytic leukaemia (CLL) / small lymphocytic lymphoma (SLL), mantle cell lymphoma (MCL) and lymphoplasmacytic lymphoma (Waldenström’s macroglobulinaemia, WM).
CLL is a slow-growing blood and bone marrow cancer that accounts for approximately one in four cases of leukaemia. Most CLL patients experience disease progression despite initial response to therapy and may require additional treatment. SLL is a clinically similar disease localized to the lymph nodes. MCL is an aggressive non-Hodgkin’s lymphoma (NHL) typically associated with very poor outcomes. MCL represents around 5% of all NHLs. The name comes from the fact that the tumour cells originate in the mantle zone of the lymph node. WM is a rare, slow-growing cancer predominantly affecting older individuals, with a mean age of 60 at diagnosis.vii, viii and median survival from five to nearly eleven years. vii, viii
The COMP adopts an opinion on Orphan Drug Designation, after which the opinion is submitted to the European Commission (EC) for endorsement. Orphan Drug Designation is a status assigned to a medicine intended for use in rare diseases.ix To be granted orphan status by the EC, a medicine must be intended for the treatment, prevention or diagnosis of a disease that is life threatening and has a prevalence of up to five in 10,000 in the European Union. Additionally, the intended medicine must aim to provide significant benefit to those affected by the condition. Orphan status provides companies with development and market exclusivity incentives for designated compounds and medicines.
In addition to ongoing Phase II/III trials in CLL, MCL and WM, acalabrutinib is currently being tested in Phase I/II trials in monotherapy as well as in combination with immunotherapy or chemotherapies in a range of other blood cancers and solid tumours.
