AOP Orphan announces EU Marketing Authorization for BESREMi® (Ropeginterferon alfa-2b) for rare blood cancer (PV)

Vienna (pts022/20.02.2019/15:20) * BESREMi® is approved as first-line monotherapy in adults for the treatment of Polycythaemia vera (PV) without symptomatic splenomegaly (1)

* BESREMi® showed high and durable hematologic response, molecular response and disease modifying capabilities without leukemogenic or carcinogenic potential (2),(3)

* BESREMi® was safe, well tolerated and will be available as a pen for self-administration for patient convenience (4)

AOP Orphan Pharmaceuticals AG (AOP Orphan), a European pioneer in rare disease, announced today that the European Commission (EC) has granted Marketing Authorization for BESREMi® (Ropeginterferon alfa-2b) as first line monotherapy in adults for the treatment of Polycythaemia Vera (PV) without symptomatic splenomegaly.

BESREMi® is now approved for use in all twenty-eight-member states of the European Union, as well as in Iceland, Liechtenstein and Norway.

The EC approval was based on data generated in the clinical development program, comprising of pivotal PROUD-PV, PEN-PV, PEGINVERA and the ongoing trial CONTINUATION-PV. Clinical study data on patients receiving treatment up to seven years are available underlining the long experience with BESREMi® in PV.

"BESREMi® is the first approved interferon in PV and now, our next goal is to make it available to patients living with this condition in Europe as soon as possible," said Andreas Steiner, Chief Executive Officer of AOP Orphan.

About BESREMi®
BESREMi® is a novel, long-acting, mono-pegylated proline interferon (ATC L03AB15). Its pharmacokinetic properties offer a new level of tolerability. BESREMi® is designed to be self-administered subcutaneously with a pen once every two weeks, or monthly during long-term maintenance. This treatment schedule is expected to lead to overall better safety, tolerability and adherence compared to conventional pegylated interferons.

For the EC Summary of Product Characteristics please visit:
http://ec.europa.eu/health/documents/community-register/html/h1352.htm

Ropeginterferon alfa-2b was discovered by PharmaEssentia Corp. based in Taiwan (see www.pharmaessentia.com). In 2009, AOP Orphan and PharmaEssentia entered into a License and Manufacturing agreement for Ropeginterferon alfa-2b which is manufactured by PharmaEssentia in its new Taichung Plant opened in Oct 2012 which has also received a GMP-certificate by a European regulatory body in Jan 2018. The finished product was developed as a pre-filled pen by Vetter (Germany) and Ypsomed (Switzerland) under the overall project lead of AOP Orphan in order to make administration easier compared to a vial or syringe. AOP Orphan has the exclusive rights for clinical development and commercialization of Ropeginterferon alfa-2b in PV, other myeloproliferative neoplasia (MPN) and chronic myeloid leukemia (CML) for European, Commonwealth of Independent States (CIS), and Middle Eastern markets. AOP Orphan informs separately when BESREMi® will be available on the respective markets.

About Polycythemia Vera
Polycythemia Vera (PV) is a rare cancer of the blood-building stem cells in the bone marrow resulting in a chronic increase of red blood cells, white blood cells and platelets. This condition increases the risk for circulatory disorders such as thrombosis and embolism, its symptoms lead to a reduced quality of life and on the long run may progress to myelofibrosis or transform to leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to blood-building stem cells in the bone marrow with a set of acquired mutations, the most important being a mutant form of JAK2 that make up the malignant clone. Important PV treatment goals are to achieve healthy blood counts (hematocrit below 45%), improve quality of life and to slow or delay the progression of disease. (5)

About AOP Orphan Pharmaceuticals AG
AOP Orphan is a multinational company with headquarters in Vienna, Austria focusing on clinical development, marketing and distribution of medicines for rare and complex diseases. AOP Orphan provides innovative treatment options, further individualized and customized services to meet physicians and patients needs across Europe, the Middle East & Asia. Currently, AOP Orphan is specializing in therapeutic solutions for HematoOncology, Cardiology & Pulmonology and Neurology & Metabolic Disorders.

To stay updated, follow AOP Orphan on LinkedIn: https://www.linkedin.com/company/aop-orphan-pharmaceuticals/

Contact
AOP Orphan Pharmaceuticals AG
Wilhelminenstrasse 91/IIf, 1160 Vienna, Austria
publicaffairs@aoporphan.com

References

(1) Summary of Product Characteristics 15.02.2019

(2) H Gisslinger, C Klade, P Georgiev, D Krochmalczyk, L Gercheva-Kyuchukova, M Egyed, V Rossiev, P Dulicek, A Illes, H Pylypenko, L Sivcheva, J Mayer, V Yablokova, K Krejcy, B Grohmann-Izay, HC Hasselbalch, R Kralovics, and JJ Kiladjian. Evidence for Superior Efficacy and Disease Modification after Three Years of Prospective Randomized Controlled Treatment of Polycythemia Vera Patients with Ropeginterferon Alfa-2b Vs. HU/BAT. 579, ASH 2018

(3) E Verger, J Soret-Dulphy, N Maslah, L Roy, J Rey, Z Ghrieb, R Kralovics, H Gisslinger, B Grohmann-Izay, C Klade, C Chomienne, S Giraudier, B Cassinat & JJ Kiladjian. Ropeginterferon alpha-2b targets JAK2V617F-positive polycythemia vera cells in vitro and in vivo. Blood Cancer Journal volume 8, Article number: 94 (2018)

(4) H Gisslinger, B Grohmann-Izay, P Georgiev, A Skotnicki, L Gercheva-Kyuchukova, M Egyed, V Rossiev, P Dulicek, A Illes, H Pylypenko, L Sivcheva, J Mayer, HC Hasselbalch, C Klade, JJ Kiladjian. FINAL RESULTS FROM PEN-PV STUDY, A SINGLE-ARM PHASE 3 TRIAL ASSESSING THE EASE OF SELF-ADMINISTRATING ROPEGINTERFERON ALFA-2B USING A PRE-FILLED PEN IN POLYCYTHEMIA VERA PATIENTS. EHA Learning Center. Gisslinger H. May 18, 2017; 182767

(5) RA Mesa, CB Miller, M Thyne, J Mangan, S Goldberger, S Fazal, X Ma, W Wilson, DC Paranagama, DG Dubinski, A Naim, S Parasuraman, J Boyle, JO Mascarenhas. Differences in Treatment Goals and Perception of Symptom Burden Between Patients With Myeloproliferative Neoplasms (MPNs) and Hematologists/Oncologists in the United States:Findings From the MPN Landmark Survey, Mesa R et al, Cancer 2017;123:449-458, DOI:10.1002/cncr.30325

Aussender:	AOP Orphan Pharmaceuticals Aktiengesellschaft
Ansprechpartner:	AOP
Tel.:	+43 503 72 44 24
E-Mail:	olena.weissenbacher@aoporphan.com
Website:	www.aoporphan.at