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Vienna, December 14, 2021:  AOP Orphan Pharmaceuticals GmbH (AOP Orphan) announced its latest analysis on Ropeginterferon alfa-2b in patients with Polycythaemia vera (PV) from its CONTINUATION-PV study presented at the ASH 2021 Annual Meeting by Professor Heinz Gisslinger from the Medical University of Vienna, Austria.

Ropeginterferon alfa-2b is a novel, long-acting, mono-pegylated proline interferon (ATC L03AB15). It is administered once every 2 weeks initially, or monthly after stabilization of hematological parameters.

AOP Orphan has been conducting a clinical development program since 2010, including the studies PEGINVERA,  PROUD-PV and CONTINUATION-PV. CONTINUATION-PV is an open-label, multicenter, phase IIIb study assessing the long-term efficacy and safety of Ropeginterferon alfa-2b versus hydroxyurea (HU) or best available treatment (BAT) in patients with PV who previously participated in the PROUD-PV study.

AOP Orphan’s clinical development program was the basis for the marketing authorizations of BESREMi® for the treatment of Polycythaemia vera in the European Union in February 2019, in Switzerland and in Taiwan in 2020, and in Israel and Korea in 2021. In November 2021, based on AOP Orphan's clinical development program, the FDA approved BESREMi® for the treatment of Polycythaemia vera in the USA.

The presentation during the ASH 2021 Annual Meeting focused on the treatment response to Ropeginterferon alfa-2b in PV patients who were either treated naïve or pre-treated with HU:

High rates of hematological and molecular responses were sustained at 5 years in both HU-naïve and HU pre-treated patients in the Ropeginterferon alfa-2b arm, even in patients with a high baseline JAK2V617F burden of more than 50%. In contrast, in the control arm, response rates were lower in HU pre-treated patients. Although early initiation of Ropeginterferon alfa-2b treatment is thought to have the greatest disease modifying benefit in patients with PV1, these data suggest that Ropeginterferon alfa-2b is also a suitable treatment option for patients switching from HU therapy, and for those with more active disease.

“This latest presentation at the American Society of Hematology Annual Meeting is yet another example of the high scientific knowledge and development capabilities of our company.” says Dr. Rudolf Widmann, Member of the Governing Board of AOP Orphan International AG.

Professor Jean-Jacques Kiladjian, from the Saint-Louis Hospital & Université de Paris in France stated earlier this year, “Ropeginterferon alfa-2b is a valuable and safe new first line therapy for PV patients. The disease modification ability of Ropeginterferon alfa-2b treatment is suggested by the striking JAK2 molecular response, and we hope that this may translate into slowing disease progression and thus improving progression-free survival and long-term patient benefit.”

1 Kiladjian JJ, Klade C, Georgiev P, et al. Towards a potential operational cure in patients with polycythaemia vera? Results from five years’ ropeginterferon alpha-2b therapy in a randomized setting. In: HemaSphere, 2021;5:(S2). Abstract Book. Abstract nr EP1076.

About BESREMi®

BESREMi® is a novel, long-acting, mono-pegylated proline interferon (ATC L03AB15). Its unique pharmacokinetic properties offer a new level of tolerability. BESREMi® is designed to be conveniently self-administered subcutaneously with a pen 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.

For the EMA Summary of Product Characteristics please visit: BESREMi®

Ropeginterferon alfa-2b was discovered by PharmaEssentia Corp., a long-term partner of AOP Orphan. In 2009, AOP Orphan in-licensed 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 Polycythaemia vera

Polycythaemia 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 in 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.

AOP Orphan Pharmaceuticals GmbH is an international pharmaceutical company with its registered office in Vienna and a focus on special diseases with a complex management. Over the past 25 years, the company has become an established provider of holistic therapy solutions from its headquarters in Vienna. This development has been made possible by a continually high level of investment in research and development on the one hand and a highly consistent and pragmatic orientation towards the needs of all our stakeholders on the other - especially the patients and their families but also the doctors and care professionals treating them.

Contact 

AOP Orphan Pharmaceuticals GmbH 
Leopold-Ungar-Platz 2, 1190 Vienna, Austria 
Dr. Christoph Klade, Chief Scientific Officer 
christoph.klade@aoporphan.com