COMP Recommends That Alprolix Maintains Orphan Designation For Treatment Of Hemophilia B

Swedish Orphan Biovitrum AB (publ) (Sobi(TM)) (STO:SOBI) and collaboration partner Biogen have received a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) recommending the European Commission (EC) to maintain the orphan designation for Alprolix® (eftrenonacog alfa), a recombinant factor IX Fc fusion protein therapy for the treatment of haemophilia B. The COMP's recommendation is now referred to the EC which is responsible for granting marketing authorisation for medicines in the EU.

An application for marketing authorisation was submitted to EMA in June 2015. In February 2016, Sobi and Biogen received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of EMA for Alprolix. If approved and the orphan designation is confirmed, Alprolix will remain on the Community Register of Orphan Medicinal Products and receive orphan designation.

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About orphan designation

To be granted orphan designation in the EU, the new medicine must be for the treatment of a life-threatening or chronically debilitating condition that affects no more than five in 10,000 people in the EU and for which no satisfactory treatments exist or, where they do exist, the new medicine will be of significant benefit to those affected by that condition. It can also be granted if a new medicine is not expected to make a sufficient return to justify the investment. A confirmed designation as an orphan medicinal product within EU may confer benefits to the product, including a 10-year period of market exclusivity from similar products following marketing authorisation.

About Haemophilia B

Haemophilia B is caused by having substantially reduced or no factor IX activity, which is needed for normal blood clotting.[1] The World Federation of Hemophilia estimates that approximately 28,000 people are currently diagnosed with haemophilia B worldwide.[2]

People with haemophilia B may experience bleeding episodes in joints and muscles that cause pain, decreased mobility and irreversible joint damage. In the worst cases, these bleeding episodes can cause organ bleeds and life-threatening haemorrhages. Infusions of factor IX temporarily replace clotting factors necessary to resolve bleeding and, when used prophylactically, to prevent new bleeding episodes.1

About Alprolix®

Alprolix® (eftrenonacog alfa) is a recombinant clotting factor therapy developed for haemophilia B by fusing factor IX to the Fc portion of immunoglobulin G subclass 1, or IgG1 (a protein commonly found in the body). This enables Alprolix to use a naturally occurring pathway to prolong the time the therapy remains in the body. While Fc fusion has been used for more than 15 years, Sobi and Biogen are the first companies to utilise it in the treatment of haemophilia.

Alprolix is currently approved for the treatment of haemophilia B in the U.S., Canada, Japan, Australia and New Zealand, where it was the first haemophilia B therapy approved to provide prolonged protection from bleeds. As with any infused protein, allergic type hypersensitivity reactions and development of inhibitors may occur following administration of Alprolix.

Sobi and Biogen are collaboration partners in the development and commercialisation of Alprolix for haemophilia B. Sobi has final development and commercialisation rights in the Sobi territory, essentially Europe, North Africa, Russia and most Middle Eastern markets. Biogen leads development and manufacturing for Alprolix and has commercialisation rights in North America and all other regions in the world excluding the Sobi territory.

About Sobi(TM)

Sobi(TM) is an international speciality healthcare company dedicated to rare diseases. Our mission is to develop and deliver innovative therapies and services to improve the lives of patients. The product portfolio is primary focused on Haemophilia, Inflammation and Genetic diseases. We also market a portfolio of speciality and rare disease products for partner companies across Europe, the Middle East, North Africa and Russia. Sobi is a pioneer in biotechnology with world-class capabilities in protein biochemistry and biologics manufacturing. In 2015, Sobi had total revenues of SEK 3.2 billion (USD 385 M) and approximately 700 employees. The share (STO:SOBI) is listed on NASDAQ OMX Stockholm. More information is available at www.sobi.com.

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