Beyond Borders: Global biotechnology report 2010

Products and pipeline
Steady growth
In many ways, 2009 looked much like
2008 in terms of product and pipeline
developments. The US Food and Drug
Administration (FDA) approved 29 new
molecular entities (NMEs) and biologic
license applications (BLAs) — about
the same number as in 2008, when
27 were approved. Approvals by the
European Medicines Agency (EMA) also
remained relatively flat. The number of
drug candidates in the clinical pipelines
of European companies increased by
16% during the year — also on par with
the growth seen in recent years. Phase
II programs led the growth, with a 22%
increase over 2008.
United States
Approvals: steady progress
After years of insufficient staffing levels,
the FDA launched a hiring initiative in
2008 to increase headcount by 1,300 new
employees. The FDA was increasing staffing
levels not only to improve the percentage
of reviews in which it meets priority and
standard review deadlines — which according
to a study in Nature were 69% and 83% in
2009, respectively — but also to meet its
responsibility to regulate tobacco starting in
June 2009. However, 2009 was also a year
with a massive pandemic scare that required
regulatory agencies to respond rapidly with
fast-track approval for an H1N1 vaccine.
Also, a lag time exists between the time a
new employee joins the FDA and comes fully
up to speed in a way that could impact the
overall performance goal of meeting 90%
of the priority and standard review times.
Considering those factors, there is reason to
hope that the hiring increase could translate
into faster approval times in years ahead.
After reaching an all-time low in 2007, FDA
drug approval numbers have rebounded
slightly in 2008 and 2009. In 2009,
the FDA approved 29 new products,
including 25 NMEs and BLAs approved
by the Center for Drug Evaluation and
Research (CDER) and four BLAs approved
by the Center for Biologics Evaluation and
Research (CBER). (These totals do not
include vaccines approved by CDER or
CBER.) Products granted approval in 2009
were in therapeutic categories that have
traditionally led product approvals, including
five approvals in cancer and four approvals
for neurological disorders.
A number of the products approved were
developed by US biotechnology companies.
For instance, in the case of Fanapt, approved
in May 2009 for the treatment of
schizophrenia, Novartis acquired the US
and Canadian commercialization rights
from Vanda Pharmaceuticals in October.
The drug had previously changed hands
numerous times. (For more details, refer to
the Deals article.) In June, AMAG gained
marketing approval for Feraheme, its
treatment of iron deficiency anemia (IDA) in
adult patients with chronic kidney disease.
Before approving it in 2009, the FDA
had twice delayed Feraheme (in October
2008, through a complete response letter
requesting additional clinical information
and the addressing of certain manufacturing
deficiencies, and again in December 2008).
The second half of the year was very
productive for biotech company approvals.
In September, Ista Pharmaceuticals’
ophthalmic solution, Bepreve, was
approved for ocular itching associated with
allergic conjunctivitis. That same month,
Allos Therapeutics gained approval for
its relapsed peripheral T-cell lymphoma
86 Beyond borders Global biotechnology report 2010
therapy, Folotyn, while Theravance gained
approval for its treatment of complicated
skin and skin structure infections (cSSSIs),
Vibativ. In November, Dyax Corporation
received FDA approval for its drug
Kalbitor — a competing product to Lev
Pharmaceuticals’ 2008 approved product,
Cinryze — for sudden attacks of hereditary
angioedema (HAE).
Success: pipeline surprises
Two biotech companies, Dendreon and
Human Genome Sciences, received positive,
late-stage clinical results for their drug
candidates that resulted in sharp upticks in
their stock performance in 2009. (See this
year’s financing article, “A higher bar,” for
more information).
The FDA had initially rejected Dendreon’s
Provenge in 2007 because of insufficient
clinical data. This experimental prostate
cancer therapy is designed to activate a
patient’s own immune system by taking
cells from a patient’s tumor, incorporating
them into a vaccine and injecting the cells
back into the patient to elicit an immune
response. The rejection was issued despite
an overwhelming recommendation from
an outside advisory panel for the drug’s
approval. With patients left with limited
choices for advanced prostate cancer that
has spread outside the prostate gland,
Dendreon continued work on Provenge and
completed an additional Phase III study.
In April 2009, Dendreon announced the
results of the study, which demonstrated
median survival rates had been extended
by about four months. The positive news
gave the company a major boost with
investors, as its market value skyrocketed
from US$440 million (US$4.62/share) at