Octapharma USA is a division of Octapharma AG, one of the largest producers of human blood plasma protein products and recombinant equivalents in the world. Established in 1983, Octapharma's mission is to provide patients with protein therapies derived from human blood plasma or produced through recombinant technologies, prioritizing patient safety. Octapharma entered the US market in 2004 and continues to grow its portfolio of products for conditions like hemophilia and immune disorders.

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Octapharma USA is a division of Octapharma AG, one
of the largest producers of human blood plasma protein
products and recombinant equivalents in the world, head-
quartered in Lachen, Switzerland. Established 31 years
ago, the “octa” in the company’s name is taken from the
Greek word for eight, the blood clotting factor deficient in
people with a bleeding disorder called hemophilia A, and
one of Octapharma’s primary clinical focus areas.
“Protein therapies have increased dramatically in num-
ber and frequency of use since the introduction of human
insulin, the first recombinant protein therapeutic, more
than 30 years ago,” says Flemming Nielsen, President of
Octapharma USA, which entered the American market in
2004 and is based in Hoboken. “Once a rarely used medical
treatment, protein therapies today have a vital role in almost
every field of medicine and are growing substantially.”
Many human diseases can be traced to quantitative
and qualitative deficiencies of proteins, which are found
not only in blood plasma, but also in every living cell, tis-
sue and organ. Some of the essential proteins in the body
ensure bleeding control, whereas others control physi-
ological reactions or fight off bacteria and viruses.
In 1983, when Chairman Wolfgang Marguerre
founded Octapharma, which today has more than 6,000
employees globally, his vision was to create a company
that would provide patients with protein therapies that
set the benchmark for safety and quality.
“Patients have always come first,” notes Nielsen, also
a member of the Management Board of Octapharma AG.
“In the end, doing what is right for patients has always
turned out right for Octapharma.”
Octapharma has been committed to patient care and
medical innovation since its conception, determined to
bring patients and the medical community life-enhancing
and lifesaving therapies. Today, Octapharma continues
to support scientific collaboration in many ways—all with
the goal of improving patient quality of life.
“Octapharma’s unique approach, from human to human,
has always been the patient-centered standard that sets us
apart,” according to Nielsen. “Our mission—for the safe
and optimal use of human proteins—emphasizes our belief
that therapies derived from human blood plasma and
human cell-lines can produce optimal patient benefits.”
Octapharma has continuously developed evermore
sophisticated technologies in order to provide effec-
tive biopharmaceuticals. The underlying principle for
Octapharma’s R&D program is to create therapies
based on human proteins, either purified from human
blood plasma or produced by recombinant technologies
applied to human cell-lines.
“Human proteins have been developed over millions of
years of evolution, and nature has done a very thorough job
of optimizing their efficiency and reducing side effects,”
Nielsen notes. “Octapharma is focused on bringing these
proteins to patients in the form of lifesaving therapeutics
in a way that respects the integrity of the proteins, but at
the same time avoids potential virus transmission.”
Patients in more than 100 countries are treated with
Octapharma therapies for Hematology (bleeding disor-
ders), Immunotherapy (immune disorders) and Critical Care
(emergency medicine). The U.S. is a main area of Octaphar-
ma’s growth, and the company’s long-term goal is to license
the complete Octapharma product portfolio in America.
“We are busy preparing for several anticipated product
launches by expanding our staff, bringing on more per-
sonnel with the key relationships needed to ensure our
continued success,” according to Nielsen. “We are also
increasing our presence at important medical congresses
and patient meetings, extending the leadership role that
Flemming Nielsen, right, Octapharma USA President and a Member of the Octapharma Management Board, notes that “Octapharma’s unique approach, from human to human, has always been the patient-
centered standard that sets us apart.”
Octapharma USA, headquartered at the Waterfront Corporate Center in Hoboken, entered the U.S. marketplace in 2004 and has consistently grown marketshare every year. The Swiss company hopes to bring
the full Octapharma product portfolio to the U.S.
has been critical to Octapharma’s international success.
It’s an extremely exciting time in the company’s history,
particularly in the U.S.”
Just this month, Octapharma USA submitted a Biologi-
cal License Application (BLA) to the U.S. Food and Drug
Administration (FDA) for one of its most novel protein
therapies to date—the first purely human recombinant
clotting factor VIII (FVIII) from a human cell-line designed
for the treatment of hemophilia A patients. The FDA
submission followed a recent recommendation from the
European Union’s central regulatory body, the Committee
for Medicinal Products for Human Use (CHMP), medical
advisors to European Medicines Agency (EMA), which
adopted a positive opinion toward Octapharma’s human
cell-line recombinant human FVIII. CHMP recommended
marketing authorization for the treatment and prophylaxis
of bleeding (also during and after surgery) in both pediatric
and adult patients with hemophilia A (congenital FVIII
deficiency). Although Octapharma is optimistic about the
success of this product in the U.S., there is no connection
between the FDA submission and the endorsement of
Octapharma’s human cell-line FVIII in Europe.
Recombinant proteins are produced in cultured human
cells. Human recombinant proteins produced in human
cell-lines show identical properties to the same protein
derived directly from human tissues and blood plasma.
They are, therefore, assumed to show improved tolerance
in patients compared to currently available recombinant
products derived from animal cell-lines.
“There is an increasing interest in a recombinant
FVIII concentrate derived from a human cell-line,” says
Nielsen. “The development of inhibitors remains one
of the major challenges in hemophilia care. Octapharma
looks forward to contributing to the unmet patient needs
in this respect, as the results from clinical trials accumu-
lated thus far look promising.”
Octapharma was among the first to develop human
proteins based on recombinant technologies and human
cell-lines. The company’s new 110,000-square-foot R&D
facility in Heidelberg, Germany, which opened in 2012,
is just one example of the company’s continuing commit-
ment to this science.
Octapharma owns five state-of-the-art production
facilities in Austria, France, Germany, Sweden and
Mexico, and continues to invest in its production
facilities with a focus on projects to increase plasma
fractionation capacity. In the U.S., which represents
approximately 50 percent of the global plasma market,
Octapharma is this year investing significantly into
building a state-of-the-art facility in Charlotte, N.C.,
which will house a plasma-testing laboratory, plasma
storage facility and the new headquarters of Octapharma
Plasma Inc., a subsidiary that operates 45 U.S. plasma
collection centers across the country.
“Octapharma did not enter the U.S. until 2004, but we
have successfully grown market share each year,” explains
Nielsen. “The company’s success is mainly due to the col-
laborations we have established with the patient and clini-
cal communities. We look forward to growing our product
portfolio in the U.S. and providing patients with novel
therapies that demonstrate Octapharma’s commitment to
the absolute highest standard of medical care.”
Octapharma USA
121 River St., Suite 1201
Hoboken, NJ 07030
Phone: (201) 604-1130
Fax: (201) 604-1131
Email: uscustomerservice@octapharma.com
Web: www.octapharma.com
No. of Employees: 70
Top Executive:
Flemming Nielsen, President of Octapharma USA/
Board Member of Octapharma AG
Products/Services:
Hematology (coagulation disorders);
Immunotherapy (immune disorders);
Critical Care (emergency medicine)
Year Founded: 2004
Creating Novel Human Protein Therapies with a Commitment to Patient Safety
Human proteins have been developed over
millions of years of evolution, and nature has done
a very thorough job of optimizing their efficiency
and reducing side effects.