● AFSTYLA®, for Hemophilia A treatment, Receives Positive Opinion from European Medicines Agency CHMP, the start of countdown to the EU marketing approval
- CHMP positive opinion would move AFSTYLA one step closer to approval in the European Union entering USD 3.6 billion Hemophilia A market
- In addition to the initiation of commercialization in the US last May, AFSTYLA’s market is expected to be expanding worldwide including European and Australian markets
- Royalty income will be secured from global sales
On Nov. 15, SK chemicals (CEO, Mahn Hoon Park) announced that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorization for AFSTYLA.
Among the new biological products that are originated from Korean biotechnology industry, AFSTYLA is the first biological product that received a CHMP’s positive opinion. CHMP’s positive opinion would move AFSTYLA one step closer to the launching into European market. In general it is expected to receive the final marketing authorization within 1 to 2 months after receiving the CHMP’s positive opinion.
AFSTYLA was invented by SK chemicals’ researchers and out-licensed to CSL, an Australian company in 2009. Since then, CSL has played major roles in the global developments including clinical trials, manufacturing process development, and license application and regulatory registration. U.S. Food and Drug Administration (FDA) approved AFSTYLA in May, 2016, which enables AFSTYLA to enter into Hemophilia A market in US. This is the first case of global commercialization through licensing-out biopharmaceuticals originated from Korean domestic technology to a global biotechnology company. AFSTYLA is currently under regulatory review process for marketing authorization in Switzerland, Australia, etc., in addition to Europe.
AFSTYLA is a recombinant Human Coagulation Factor VIII specially designed as the first and only single-chain Factor VIII structure for hemophilia A treatment. As a single chain structure form of Factor VIII, AFSTYLA provides the novel treatment option of twice weekly dosing and has greater molecular stability, compared to other recombinant Factor VIII products which are composed of two heavy and light chains in separately associated structure.
Furthermore, AFSTYLA demonstrated an excellent safety profile with no inhibitor development in previously treated patients undergoing prophylaxis which is the major complication of Factor VIII replacement therapy in hemophilia A patients.
CSL expects that AFSTYLA could be more competitive in the global hemophilia A market as an innovative and effective therapy. Accordingly, SK chemicals expects to receive royalty from the sales.
According to Datamonitor (www.datamonitor.com), the European hemophilia A market size reached about 3.6 billion USD, and the whole global market size was 7.2 billion USD as of year 2015. The value of hemophilia A market would continue to grow and the global market is expected to reach 8.3 billion USD, about a 17 % increase, by year 2020.
[Definition of the Terms]
1) Hemophilia A
Primarily affecting males, hemophilia A is a congenital bleeding disorder characterized by deficient or defective factor VIII. People with hemophilia A may experience prolonged or spontaneous bleeding, especially into the muscles, joints or internal organs. According to the United States Centers for Disease Control and Prevention (CDC), the condition affects approximately 1 in 6,000 male births.
2) AFSTYLA
Abbreviation for “Affinity Style Long Acting”. Means anti-hemophilic drug, safe and providing longer-lasting efficacy with its high covalent bond within the body.
3) An essential blood-clotting protein, Factor VIIIOne factor involving blood clotting in 13 blood-clotting proteins. Hemophilia A is a genetic deficiency in Factor VIII.
[About the company]
CSL Limited (www.csl.com.au)
CSL founded in 1916 is a leading global biotherapeutics company with a dynamic portfolio of life-saving innovations, including those that treat hemophilia and immune deficiencies, as well as vaccines to prevent influenza. It — including its two businesses CSL Behring and Seqirus — operates in over 30 countries with more than 16,000 employees.