● The Start of Countdown to the Marketing Approval of Its Anti-Hemophilia Agent as the First Domestic Biopharmaceutical R&D Achievement to Be Sold in the US and EU Markets

- The completion of the application of NBP601 for EMA marketing authorization following the submission for FDA BLA in July
- The first domestic biopharmaceutical R&D performance which reaches to global market



The countdown for the global commercialization of SK chemicals’ out-licensed anti-hemophilia agent starts.

On 23, SK chemicals (CEO Mahnhoon Park) announced that its partner CSL has submitted its Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its novel biological product ‘NBP601 (CSL627)’ for the treatment of hemophilia A.

‘NBP601’ will become the first outcome of the Korean domestic R&D achievement which applies for marketing approval in global markets including the US and EU markets.

‘NBP601’, a novel recombinant therapeutic protein for hemophilia A treatment, was invented by SK chemicals’ scientists and technology and out-licensed to CSL, an Australian company, for the global development in 2009. After IND approval in 2011, global clinical trials have been worldwide conducted.

‘NBP601’ is a novel recombinant Factor VIII protein with improved intrinsic stability and increased binding to von Willebrand Factor, leading to a longer-lasting therapeutic effect compared to a full length Factor VIII. NPB601 met all primary endpoints in global clinical trials for EMA approval and more than 30% of patients on prophylaxis could be maintained on a twice weekly dosing frequency.

Results from the phase I/III study presented earlier this year at the International Society on Thrombosis and Haemostasis (ISTH) congress in Toronto showed that patients treating prophylactically had a median annualized bleeding rate (ABR) of 1.14 and a median annualized spontaneous bleeding rate (AsBR) of 0.00.

The data also showed that, of 848 bleeds treated in the study, 94 percent were controlled with no more than two infusions of ‘NBP601’, with 81 percent controlled by one infusion. Moreover, hemostatic control of a bleeding event treated with ‘NBP601’ was assessed by the investigator as excellent or good 94 percent of the time (835 assessed bleeding events). No patient in the clinical study developed an Inhibitor reaction, one of the most severe complications of factor replacement therapy in hemophilia A.

SK chemicals announces that it will devote a company-wide effort to the successful global launch of ‘NBP601’ with close cooperation with CSL Behring.

Dr. Hun-Taek Kim, director of SK chemicals Innovative R&D Center, who is the main inventor and project leader of NBP601 project said, “The submission of Marketing Authorization application (MAA) to the EMA following BLA submission for FDA in last July shows that R&D capability of SK chemicals has been globally acknowledged.” He added, “We will continue to develop therapeutics for the treatment of the diseases with unmet needs.”

Hemophilia A is a congenital bleeding disorder characterized by deficient or defective factor VIII. People with hemophilia A may experience prolonged or spontaneous bleeding due to the lack of blood coagulation factors by genetic deficiency. About 1 in 5,000 to 10,000 males suffer from hemophilia.

The current main treatment for hemophilia A, Factor VIII has formed a market size of $6.4 billion in 2013 and the sales of recombinant products have grown to $5.1 billion in 2013 from $3.9 billion in 2008 which equals to 5.9% annual growth (Source: MRB Report *** and publications from R&D pipeline news La Merie).

A big first-mover advantage upon its approval is expected since the MAA submissions of long-acting products being developed by other major players in anti-hemophilia industry such as Baxter and Bayer have not been announced.



[Photo]
NBP601, Korea’s first bio-based new drug to treat hemophilia; applications for approval have been made in the United States and Europe.



[Definition of Terms]

1.International Society on Thrombosis and Haemostasis (ISTH)
ISTH is a prestigious global organization established in 1954 that has over 3,000 members from more than 80 countries and studies thrombotic and bleeding disorders.

2.MRB Report
The syndicated reports published by The Marketing Research Bureau which is an independent market research firm that investigates in blood derivatives and biopharmaceutical markets.

3. ABR, annualized bleeding rate
The number of treated bleeding events within the evaluation period, extrapolated to the expected number of treated bleed in a year.

4.AsBR, annualized spontaneous bleeding rate
The number of treated spontaneous (non-traumatic) bleeding events within the evaluation period, extrapolated to the expected number of treated spontaneous (non-traumatic) bleed in a year.

5. Inhibitor reaction
Pathological formation of neutralizing antibodies that removes or reduces the effect of a drug.


[Company Introduction]

CSL Limited (www.csl.com.au)
CSL is a biotherapeutics company with a portfolio of plasma-derived proteins, recombinant proteins, vaccines and etc. It was founded in 1916 and its headquarters is located in Melbourne, Australia. CSL operates in over 30 countries in Europe, North America and Australia with more than 14,000 employees. It owns large manufacturing sites in Australia, Germany, Switzerland and the United States. The total sales in 2012 were approximately $5.5 billion.