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Vienna, Austria (pts008/17.12.2018/10:20) - AOP Orphan's EMA marketing authorization application for Ropeginterferon alfa-2b/BESREMi® resulted in a positive opinion adopted by EMA's CHMP. The European Commission will review the CHMP recommendation and usually delivers its final decision within approximately two months

- BESREMi® is indicated as a monotherapy in adults for the treatment of Polycythaemia vera without symptomatic splenomegaly

- BESREMi® has been shown to induce complete hematologic and high clinical response rates with good tolerability, as well as high molecular response rates and disease modifying capabilities, which may result in a delay of disease progression

- BESREMi® will be available as a pen for patient self-administration

Vienna, 17th of December 2018: AOP Orphan Pharmaceuticals AG (AOP Orphan) today announced that EMA´s CHMP adopted a positive opinion for approval of Ropeginterferon alfa-2b/BESREMi® indicated as monotherapy in adults for the treatment of Polycythaemia vera without symptomatic splenomegaly.

Ropeginterferon alfa-2b/BESREMi® is a novel, long-acting interferon, which is administered once every two weeks, or monthly after stabilization of hematological parameters. This treatment schedule is expected to lead to overall better safety, tolerability and adherence compared to conventional pegylated interferons.

AOP Orphan has been running the BESREMi® clinical development in PV since 2010. The latest phase III data, three years treatment, and phase II data, up to seven years treatment, were presented at ASH 2018. In summary, BESREMi® showed high hematologic and clinical response rates with good tolerability.

In addition, BESREMi® showed high molecular response rates, associated with the ability to reduce allelic burden of both mutant JAK2 and importantly also non-JAK2 mutations, which are believed to have a role in disease progression.

Andreas Steiner, Chief Executive Officer of AOP Orphan commented: "Although interferons are a treatment modality widely used throughout the myeloproliferative neoplasms including CML, BESREMi® will be the first licensed interferon in any of these indications. Physicians experienced in the management of the disease and administration of BESREMi® during the clinical studies expect many advantages to patients with PV."

About Ropeginterferon alfa-2b/BESREMi®
Ropeginterferon alfa-2b/BESREMi® is a novel, long-acting, mono-pegylated proline interferon (ATC L03AB15) with improved pharmacokinetic properties offering improved tolerability and adherence to treatment. It is administered once every two weeks, or monthly during long-term maintenance, and is expected to be the first interferon approved for PV worldwide.

Ropeginterferon alfa-2b was discovered by PharmaEssentia, a long-term partner of AOP Orphan. In 2009, AOP Orphan has in-licensed from PharmaEssentia Corporation the exclusive rights for clinical development and commercialization of Ropeginterferon alfa-2b in PV, other MPNs and CML for European, Commonwealth of Independent States (CIS), and Middle Eastern markets.

About Polycythemia Vera
Polycythemia Vera (PV) is a cancer of the blood-building cells in the bone marrow resulting in a chronic increase of red blood cells, white blood cells and platelets. This condition may result in circulatory disorders such as thrombosis and embolism, as well as malignant transformation to myelofibrosis or leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to a set of acquired mutations, the most important being a mutant form of JAK2 that make the malignant clone.

About AOP Orphan Pharmaceuticals AG
AOP Orphan is a multinational company with headquarters in Vienna, Austria focusing on clinical research, development and distribution of medicines for rare and complex diseases. The company also provides individualized and customized services to meet and accommodate the needs of physicians and patients across Europe, the Middle East & Asia. Currently AOP Orphan is concentrating on orphan and complex diseases in HematoOncology, Cardiology & Pulmonology and Neurology & Metabolic Disorders.

Contact
AOP Orphan Pharmaceuticals AG
Wilhelminenstrasse 91/IIf, 1160 Vienna, Austria
Dr. Christoph Klade, Chief Scientific Officer
E: christoph.klade@aoporphan.com

Submitter: AOP Orphan Pharmaceuticals Aktiengesellschaft Contact: AOP Phone: +43 503 72 44-24 E-Mail: olena.weissenbacher@aoporphan.com Website: www.aoporphan.at