The US Food & Drug Administration (FDA) has issued a complete response letter for the drug BESREMi® with the active pharmaceutical ingredient, Ropeginterferon alfa-2b, in which the FDA confirms the efficacy and safety of the drug. This assessment was based on the clinical development program by the European rare disease company AOP Orphan.

On March 12, 2021, according to PharmaEssentia Corp., the FDA in the United States issued a so-called Complete Response Letter in respect to the submission of BESREMi® (Ropeginterferon alfa-2b) for the treatment of polycythaemia vera, a rare form of blood cancer.

The Complete Response Letter confirms the safety and efficacy of BESREMi® in compliance with FDA regulations.

The Complete Response Letter comes almost a year after PharmaEssentia’s application to the FDA and is based on a clinical study program by AOP Orphan, which started in Europe in 2010. AOP Orphan congratulates PharmaEssentia on achieving this important milestone.

“Today’s FDA 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, Chief Therapeutics Development Officer and Member of the Board of the Vienna-based company AOP Orphan.

This is the first of AOP Orphan’s ambitious US registration projects. This achievement speaks not only to the scientific expertise and clinical development know-how of AOP Orphan, but also to the efficacy of the product.

Final approval of BESREMi® in the US can take place after successful inspection of the manufacturing facilities of PharmaEssentia by the FDA officials. Due to the COVID-19 pandemic, they were as of yet unable to travel to Taiwan to inspect the manufacturing facilities, a requirement for any new drug approval by the FDA.

Clinical studies conducted by AOP Orphan

Ropeginterferon alfa-2b is a specifically modified pegylated Interferon alpha 2b invented by PharmaEssentia’s CEO KC Lin.

In 2009, AOP Orphan in-licensed the exclusive rights for the clinical development and commercialization of Ropeginterferon alfa-2b in polycythaemia vera and other myeloproliferative neoplasms (MPNs) for Europe, the Commonwealth of Independent States (CIS), and Middle Eastern markets.

Ever since, AOP Orphan has continually invested its hematology and drug development know-how along with considerable funds into development of an injection pen for patients’ at-home self-administration.

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 in 2020, and in Israel 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).

Polycythaemia vera patients in approximately 20 European countries are already being treated with Ropeginterferon alfa-2b. AOP Orphan is continuously working on increasing access to this life-saving drug for many more patients and continues the clinical development of BESREMi®.

Source: StreetReporters.ng

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