Aplidin granted orphan drug status for MM

The European Commission has granted PharMar's Aplidin, its second marine-derived compound in clinical development, orphan drug status for the treatment of multiple myeloma (MM) following adoption of a positive opinion by the Committee for Orphan Medicinal Products (COMP) of the European Agency for the Evaluation of Medicinal Products (EMEA).

Last September, the Food & Drug Administration (FDA) granted orphan drug status to Aplidin for the same indication in the US.

In July 2003, Aplidin was granted orphan drug status in the EU for the treatment of acute lymphoblastic leukaemia (ALL), a malignant disease of the bone marrow. Orphan drug designation in the European Union offers several incentives, such as 10-years marketing exclusivity throughout Europe for the therapeutic indication for which it was granted, protocol assistance from the EMEA to optimise drug development, reduction of fees for all types of centralised procedure activities and access to grants from EC and Member States programmes.

Multiple myeloma is the second most common haematological malignancy after Non-Hodgkin's Lymphoma and is part of a spectrum of diseases labelled Plasma Cell Dyscrasia. Plasma cells are the cells responsible for forming antibodies against bacteria and foreign agents. For reasons that are unclear, these cells lose their ability to respond to controlling signals from a hierarchy of immune cells. Plasma cells then divide with no control and form abnormal proteins, which results in damage to the bone, the kidney, and/or other organs of the body. Incidence and mortality rates are similar in Europe and in the United States, being close to 5/100,000 and 4/100,000 inhabitants respectively. It accounts for about 1% of all cancer types and for 10% of all haematological malignancies.

The survival rate for MM has not changed for the past three decades, with only about 28% of patients surviving for longer than five years. No single standard therapy currently exists for multiple myeloma patients that have relapsed or become resistant to treatment and these patients have an expected survival of just 6-9 months. Despite high-dose chemotherapy treatments and the appearance of some new agents with significant activity in the relapsed disease, MM remains an incurable disease, with 10-year survival rates estimated to be less than 5%.

Isabel Lozano, chief executive officer of PharmaMar, said: 'This orphan drug designation in Europe recognises the urgent need for new therapies for the treatment of multiple myeloma. This will support the sustained clinical development of Aplidin in MM, as well as in other haematological malignancies, which will start before the year end.'