US-based non-profit laboratory Associated Regional and University Pathologists (ARUP) has secured the CE mark for its companion diagnostic product, the AAV5 DetectCDx test.

The AAV5 DetectCDx is a companion diagnostic developed in partnership with US-based biotechnology company BioMarin.

The CDx test is intended to help identify adult patients with severe haemophilia A, who are eligible for the treatment with BioMarin’s new gene therapy, Roctavian.

AAV5 DetectCDx was already approved in Europe, with a CE mark under the previous In-Vitro Diagnostic Medical Devices Directive (IVDD), granted in August 2020.

ARUP secured the CE mark approval under the European Union’s (EU) In Vitro Diagnostic Medical Devices Regulation (IVDR), which requires high standards for patient safety.

With the IVDR approval, ARUP is allowed to test the samples from those living in the EU and other countries that recognise EU regulations, at its advanced laboratory in Salt Lake City, Utah.

ARUP clinical trials and PharmaDx vice president Jay Patel said: “We are proud to be involved in this effort to support the needs of patients with severe haemophilia A.

“We’ve demonstrated by our ability to execute on these stringent regulatory requirements that we’re committed to patient safety and access to testing.

“Very few clinical labs have the quality management system and competencies in place to execute a new companion diagnostic at this regulatory level. We are proud of our ability to deliver at the highest levels for patients and for our partners.”

BioMarin’s new gene therapy Roctavian leverages adeno-associated virus serotype 5 (AAV5) as a vector to deliver a functional copy of the gene for FVIII in a one-time infusion.

It aims to reduce or eliminate the need for chronic prophylactic treatment and is recommended only for individuals without anti-AAV5 antibodies.

The AAV5 DetectCDx is designed to detect the presence of pre-existing anti-AAV5 antibodies that can inhibit the efficacy of the treatment.

The test also received FDA approval in June this year, as the first FDA-approved companion diagnostic immunoassay for any gene therapy, said ARUP.

ARUP PharmaDx companion diagnostics team group manager Jennifer Granger said: “We are gratified to see the results of this colossal effort from both teams and to fulfil an unmet need for patients with rare diseases such as haemophilia A.”