Madrid, November 19 (ots/PRNewswire) - PharmaMar announced today
that Aplidin(R) (1), its second marine-derived compound in clinical
development, was granted orphan drug status by the European
Commission 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 United
In July 2003, Aplidin(R) was granted orphan drug status in the
European Union 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 hematological 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% (Medscape CME 2003).
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
Aplidin is a cyclic peptide, originally isolated from the marine
tunicate Aplidium albicans, currently obtained by total synthesis. It
induces apoptosis rapidly and persistently, inhibits VEGF secretion
and blocks cell-cycle.
It is currently in therapeutic exploratory clinical evaluation
(phase II) in solid and haematological malignancies, including
paediatrics. The clinical program involves hospitals in Europe,
Canada and the US. Approximately 400 patients have been treated. In
the preclinical development, human leukaemia, myeloma and lymphoma
tumour cell lines are particularly sensitive to Aplidin. There is no
evidence of cross-resistance with commonly used therapeutic agents
for haematological malignancies.
There is no clinical evidence of relevant bone marrow toxicity.
Its side effects are reversible and manageable (including muscular
and liver biochemical alterations). Hair loss and oral ulcers are
Aplidin was granted Orphan Drug Designations for the treatment of
Acute Lymphoblastic Leukaemia in the European Union in 2003 and in
the US in 2004.The US FDA also granted ODD for Multiple Myeloma in
(1) 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 Yondelis
(TM) 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 by the FDA in 2004,
and Orphan Drug for ovarian cancer by the E.C. 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, and for multiple myeloma
by the FDA in 2004; Kahalalide F in phase II and ES-285 in phase I
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
ots Originaltext: PharmaMar
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Lola Casals, PharmaMar, Tel: +34-91-846-6000. David Yates & Deborah
Scott, Financial Dynamics, Tel: +44-207-831-3113