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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."
About Aplidin(R)
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
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

Contact:

Lola Casals, PharmaMar, tel: +34-91-846-6000 or David Yates & James
Strong, Financial Dynamics, tel: +44-(0)20-7831-3113