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FDA Grants Aplidin(R) Orphan Drug Status for the Treatment of Multiple Myeloma
Madrid, Spain (ots/PRNewswire) - PharmaMar announced today that Aplidin(R), its second marine-derived compound in Phase II clinical development, has been granted orphan drug status by the United States (US) Food and Drug Administration (FDA), for the treatment of Multiple Myeloma (MM). This is the second orphan drug designation granted to Aplidin by the FDA. The first was granted in June for the use of Aplidin in the treatment of acute lymphoblastic leukaemia, a malignant disease of the bone marrow.
Orphan drug designation is awarded to drugs that offer potential therapeutic value in the treatment of rare diseases and conditions and therefore may benefit directly from the provisions of the Orphan Act which includes: regulatory assistance and numerous financial incentives for the development and approval of the orphan product, including seven years of marketing exclusivity; New Drug Application fee waivers; tax credits for clinical research and grant funding for the investigation of the rare disease treatment.
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. There are approximately 45,000 people living with multiple myeloma in the US (Multiple Myeloma Research Foundation, 2004). It is estimated that approximately 15,270 new cases of multiple myeloma will be diagnosed during 2004 in the US and that 11,070 people will die of the disease (American Cancer Society, 2004)
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% (Medscape CME 2003).
"The FDA's orphan drug designation for Aplidin represents an important milestone in Aplidin's development", said Isabel Lozano, Chief Executive Officer of PharmaMar. "We look forward to continuing with the clinical development of Aplidin as a potential treatment for MM patients, based on the early evidence of activity seen in MM and other haematological malignancies."
Aplidin is a novel antitumour agent derived from the marine tunicate Aplidium albicans. It induces rapid and persistent activation of apoptosis combined with blocking of cell division in the G1/G2 phase of the cell cycle in tumour cells. It also inhibits the secretion of vascular endothelial growth factor (VEGF), a crucial protein involved in the vascularisation and growth of a number of tumours, and the expression of the VEGF receptor 1 (VEGFR1).
Aplidin(R) is a PharmaMar registered trademark.
PharmaMar is a biopharmaceutical company, advancing cancer care through the discovery and development of innovative marine-derived medicines. PharmaMar's clinical portfolio currently includes YondelisTM in phase II clinical trials (co-developed with Johnson & Johnson Pharmaceutical Research & Development), designated Orphan Drug for STS by the European Commission (E.C.) in 2001 and Orphan Drug for ovarian cancer in 2003; Aplidin(R), in phase II, designated Orphan Drug for acute lymphoblastic leukaemia by the E.C. in 2003 and by the FDA in 2004; Kahalalide F in phase II and ES-285 in phase I clinical trials.
PharmaMar, based in Madrid, Spain, is a subsidiary of the Zeltia Group (Spanish stock exchange: ZEL.MC; Bloomberg: ZEL SM; Reuters: ZEL.MC). PharmaMar can be found on the Web at http://www.pharmamar.com
ots Originaltext: Zeltia Group
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Lola Casals, PharmaMar, tel: +34-91-846-6000 or David Yates & James
Strong, Financial Dynamics, tel: +44-(0)20-7831-3113