● Korea`s first recombinant protein medication

- Stability and half-life significantly improved… Proven through clinical trials
- Global three-phase trials concluded… FDA New Drug Application completed in July



SK chemicals (Man-hun Park, President) announced on the 29th of this month that its global partner has completed a New Drug Application (NDA) for U.S. FDA marketing authorization regarding the biopharmaceutical material NBP601, their proprietary hemophilia medication.

Developed with SK chemicals` very own technologies, NBP601 is a recombinant biopharmaceutical for hemophilia that was technology-exported to Australia`s CSL Limited in 2009, and the medication has since been undergoing global clinical trials throughout the U.S. and Europe.

This marks the first time that a recombinant protein medication developed by a Korean pharmaceutical company entered the marketing approval stage with the U.S. FDA.

NBP601 is a recombinant biopharmaceutical that dramatically improves the stability of coagulation factor VIII (FVIII), an essential blood-clotting protein also known as anti-hemophilic factor (AHF). The half-life of the drug was extended to reduce the number of required doses for improved dosing convenience in patients. All the main evaluation requirements for FDA drug approval were met through global clinical trials, and the improvement in the quality of life for patients was considered to have tremendous merit.

According to the clinical trial results publicized at the International Society on Thrombosis and Haemostasis (ISTH) Congress held recently in Toronto, patients who received prophylactic treatment had an average annualized bleeding rate (ABR) of 1.14, a 40% improvement compared to conventional drugs. The annualized spontaneous bleeding rate (AsBR) recorded an average of 0.0.

Of the total 848 clinical trial cases, bleeding was managed with two doses of NBP601 in 94% of the cases and a single dose in 81% of the cases. In regards to hemostasis management, 94% of the total 835 cases yielded an "excellent" or "good" result. Notably, not a single case of neutralizing antibody response was reported, a main side effect of hemophilia A medication.

Typically, the process to obtain final approval for FDA drug marketing authorization requires about one year. SK chemicals is planning to direct company-wide efforts toward a successful global launch of NBP601 together with its partner CSL Behring.

An SK chemicals official stated, "Through cooperation with CSL Behring, we will do our best to ensure that NBP601 is introduced to the U.S. biopharmaceutical market as smoothly as possible for quick adoption. As NBP601 is a major step forward in stability and half-life compared to existing drugs, we firmly believe that it will open a new chapter in hemophilia treatment."

Hemophilia A is a rare congenital bleeding disorder that is endemic to a factor VIII defect or deficiency, exhibiting difficulties in hemostasis due to the inability to produce blood-clotting factors in the human body caused by genetic defects. Currently, the condition affects one in every 5,000 to 10,000 men worldwide.

FVIII, the current leading form of treatment for hemophilia A, constitutes a $6B USD (approx. ₩ 6.8T) global market as of 2011. Of this figure, sales of recombinant products have grown from $4.3B USD (approx. ₩ 4.8T) in 2011 to $4.9B USD (approx. ₩ 5.5T) in 2012. (Source: MRB Report; R&D Pipeline News, La Merie Publishing)

[Glossary]

International Society on Thrombosis and Haemostasis (ISTH)
Founded in 1954, the ISTH is a prestigious international community of scientists and physicians specializing in blood-clotting disorders, with about 3,000 members spanning more than 80 countries.

Annualized Bleeding Rate (ABR)
Number of bleeding episodes per year.

Annualized Spontaneous Bleeding Rate (AsBR)
Number of natural or spontaneous bleeding episodes per year.

Neutralizing Antibody Response
An improper response that inhibits or diminishes a drug`s effect.

MRB Report
Research report published by the Marketing Research Bureau (MRB), a blood and plasma research specialist.


[Company Info]

CSL Limited (www.csl.com.au)
Founded in 1916 and based in Melbourne, Australia, CSL Limited is a biopharmaceutical company specializing in plasma-derived proteins and related recombinant proteins as well as vaccines. It employs 14,000 employees throughout 30 countries in Europe, North America, and Australia and has a large production base in Australia, Germany, Switzerland, and the U.S. In 2012, CSL Limited generated approximately $5.5B USD in revenue.