Beyond Borders: Global biotechnology report 2010

European Phase III pipeline by indication, 2009
Autoimmune
12%
Metabolic and endocrine
13%
Cancer
22%
Source: Ernst & Young, MedTRACK and company websites
European approvals
The EMA was active in approving products
for a variety of therapeutic categories,
including cancer, neurology, cardiovascular,
metabolic disorders and immunology.
Most products approved by the EMA
were from US-based companies or large
pharmaceutical organizations; however, a
handful of European-based biotechs secured
approvals as well.
Belgium-based TiGenix received EMA
approval for ChondroCelect, a cartilagerepair
therapeutic, in October. The
cell-based medicinal product consists of
chondrocytes (cartilage-forming cells)
that are taken from a healthy region of a
Neurology
11%
Other
20%
Inflammation
9%
Infectious disease
6%
Cardiovascular
7%
patient’s cartilage, grown outside the body,
and then surgically re-implanted. Belgiumbased
UCB also garnered market approval
in Europe for its drug Cimzia for rheumatoid
arthritis. Cimzia had previously gained US
marketing authorization for moderate-tosevere
Crohn’s disease.
Other approvals included Ferring
Pharmaceutical’s Firmagon in February for
patients with advanced prostate cancer.
Firmagon was previously approved by the FDA
in 2008. Orphan Europe’s product Vedrop
was approved for cholestatis, a condition
in which the flow of bile from the liver is
blocked. Victoza, a Novo Nordisk product, was
approved for type 2 diabetes in June.
90 Beyond borders Global biotechnology report 2010
European companies also received FDA
approval for a number of products in 2009.
GlaxoSmithKline received accelerated
approval in October 2009 for Arzerra,
a treatment for refractory chronic
lymphocytic leukemia. Arzerra, which
GSK obtained through a collaboration with
Genmab, is a monoclonal antibody that
causes the body’s immune response to
fight against normal and cancerous B-cells.
Azerra went on to receive conditional
approval in Europe in January 2010
for the treatment of refractory chronic
lymphocytic leukemia (CLL), but only for
the approximately 25% or so of patients
who do not respond to the standard
therapies fludarabine and alemtuzumab,
which EMA has already approved.
Lundbeck secured two approvals in 2009.
Its Sabril, for treatment of infantile spasms
in pediatric patients and refractory complex
partial seizures in adults, was approved in
August 2009. Earlier in the year, Lundbeck
licensed Atryn from GTC Biotherapeutics
and received approval for the antithrombin
deficiency medication in February.
In another licensing deal, Cypress
Bioscience licensed Savella from French
drug-maker Pierre Fabre and then signed
a commercialization agreement with
Forest Laboratories. In January, the FDA
approved the product for the treatment
of fibromyalgia. Milnacipran was originally
approved in France in 1996 for major
depression episodes but was introduced in
the United States for the first time with the
fibromyalgia indication.
Outcomes-based pricing arrangements
Facing mounting pricing pressures,
payors are seeking ways to leverage
pay-for-performance reimbursement
mechanisms. In this environment,
risk-sharing pricing arrangements — where