EMA recommends AZ/Acerta drug for orphan status in three indications
pharmafile | February 25, 2016 | News story | Research and Development | AstraZeneca, EMA, Orphan Drugs
The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has recommended AstraZeneca and Acerta’s acalabrutinib (ACP-196) for designation as an orphan medicinal product in three separate indications.
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, MG).
CLL is a slow-growing cancer of the blood and bone marrow cancer that accounts for approximately a quarter of all leukaemia cases. Most CLL patients experience disease progression despite initial response to therapy and may require additional treatment. SLL is a clinically similar disease, localised to the lymph nodes.
MCL is a rare and aggressive non-Hodgkin’s lymphoma (NHL), with generally poor outcomes; while WM is a rare, slow-growing cancer predominantly affecting older individuals. Most diagnoses occur at the age of around 60, and the median survival ranges from five to nearly eleven years.
Orphan drug status is awarded for drugs to treat life-threatening rare diseases, affecting up to five in 10,000 people in the European Union. To qualify for the status – and the benefits its brings, including development and market exclusivity incentives – a medicine must demonstrate significant benefit in treating such conditions.
Sean Bohen, executive vice president of Global Medicines Development and chief medical officer at AstraZeneca, says: “Today’s three positive opinions recommending acalabrutinib for designation as an orphan medicinal product are important milestones. They reinforce the strategic rationale for our investment in Acerta, demonstrating clear progress in developing a potential best-in-class medicine that could transform treatment for patients across a range of blood cancers.
“The positive opinions underscore the continued need for the development of new therapies in these serious and life-threatening conditions and support our commitment to bring new medicines to patients as quickly as possible.”
Joel Levy
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