28 NJBIZ Business Profiles 2014 29

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.