BESREMi® (Ropeginterferon alfa-2b) approved by the US FDA
The US Food & Drug Administration (FDA) approved BESREMi® (active pharmaceutical ingredient Ropeginterferon alfa-2b), confirming the efficacy and safety for treatment of Polycythaemia vera, a rare blood cancer disease. This approval was based on the clinical development program by AOP Orphan, the European pioneer in the field of rare and special diseases.
Vienna, November 15, 2021. In 2009, AOP Orphan in-licensed the exclusive rights for clinical development and commercialization of Ropeginterferon alfa-2b in Polycythaemia vera (PV), a rare disease, and other myeloproliferative neoplasms (MPNs) for European, Commonwealth of Independent States (CIS), and Middle Eastern markets. Based on a pivotal study development program, which included the studies PEGINVERA, PROUD-PV and CONTINUATION-PV the European Medicines Agency (EMA) authorized BESREMi® for the treatment of PV in February 2019. The whole clinical development program was designed and conducted by AOP Orphan in Europe.
PharmaEssentia Corp., AOP Orphan’s licensor, subsequently submitted the clinical data generated in the clinical development program for marketing authorisation of BESREMi® by the FDA in the United States. On March 12, 2021, according to PharmaEssentia, the FDA issued a Complete Response Letter for BESREMi® (Ropeginterferon alfa-2b) confirming the safety and efficacy of BESREMi® in compliance with FDA regulations. Now, after PharmaEssentia’s manufacturing sites in Taiwan have successfully passed the FDA’s GMP pre-approval inspection in September 2021, the FDA approved BESREMi® for sale in the United States.
“FDA’s confirmation of BESREMi®’s safety and efficacy, based on AOP Orphan’s clinical study program and scientific knowledge, attests to the quality of work at our company" says Dr. Rudolf Widmann, Member of the Governing Board of AOP Orphan International. "We are extremely happy that now patients in the US have access for treating this rare blood cancer disease. This is the first of AOP Orphan’s ambitious projects to get medicinal products for rare and special diseases approved in the US. This achievement speaks not only to the scientific expertise and clinical development know-how of AOP Orphan, but also supports our company strategy to make drugs for rare and special diseases available worldwide."
Clinical studies conducted by AOP Orphan
Ropeginterferon alfa-2b is a specifically modified pegylated Interferon alpha 2b invented by PharmaEssentia’s CEO KC Lin.
The application of this third-generation interferon to the treatment of PV was invented by Dr. Rudolf Widmann of AOP Orphan: The company has continually invested in its hematology and drug development know-how to get an injection pen for patients’ at-home self-administration approved. Several clinical studies have been conducted by the company leading to a successful marketing authorization of BESREMi® for the treatment of Polycythaemia vera in the EU in February 2019, in Switzerland and in Taiwan in 2020, in Israel and Korea in 2021.
Besides achieving high rates of complete hematologic responses including freedom of phlebotomy in 8 out of 10 patients, BESREMi® offers the possibility of disease modification and eventually operational cure in a subset of patients. This is exemplified by a decrease of mutant JAK2 allele burden (the disease-causing oncogene) from 37.3% at baseline to 7.3% in patients receiving Ropeginterferon alfa-2b, while an increase from 38.1% to 42.6% in the control group receiving hydroxyurea/best available therapy was observed (p<0.0001).1
Polycythaemia vera patients in approximately 20 European countries are already being treated with Ropeginterferon alfa-2b. AOP Orphan is continuously working on increasing access for many more patients and continues the clinical development of BESREMi®.
BESREMi® is a 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. Ropeginterferon alfa-2b was discovered by PharmaEssentia, a long-term partner of AOP Orphan.
For the EMA Summary of Product Characteristics please visit: https://www.ema.europa.eu/en/documents/product-information/besremi-epar-product-information_en.pdf
AOP Orphan Pharmaceuticals AG 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. In the third quarter of 2020, AOP Orphan took over Amomed and SciPharm, two European health care companies, thus continuing its consistent path of growth into a pan-European health care group specializing in special diseases with a complex management. Within the overall portfolio of approximately 20 products, the AOP Orphan group expects sales of approximately EUR 200 million in 2021 and intends to increase its revenues to one billion euros by 2030.
For more information, please contact:
AOP Orphan Pharmaceuticals AG
Wilhelminenstraße 91/IIf, 1160 Vienna
Ms. Nina Roth, MAS
T: +43 676 3131509