Partnership expands access in EU to Roctavian companion diagnostic test
Therapy for hem A patients who don't already possess antibodies against AAV5
A partnership between ARUP Laboratories and Medicover will improve access to AAV5 DetectCDx, the former’s companion diagnostic test to assess hemophilia A patients’ eligibility to Roctavian (valoctocogene roxaparvovec-rvox) in the European Union (EU).
The partnership, which allows testing to be done at one of Medicover’s German facilities for patients living at all European countries where Roctavian is approved, is expected to ultimately result in better access to the BioMarin Pharmaceutical’s gene therapy.
Roctavian was conditionally approved last year in the EU for adults with severe hemophilia A. It was approved in the U.S. earlier this year.
Under both approvals, Roctavian may be used in patients who don’t already possess antibodies against AAV5, the virus that carries and delivers the genetic load to patient cells, because such antibodies can make the therapy ineffective.
Developed by ARUP, a national reference lab that offers more than 3,000 tests and test combinations, in collaboration with BioMarin, the AAV5 DetectCDx test can detect the presence of these antibodies.
“We have leveraged key ARUP strengths—our expertise in assay development and our robust quality management system that allow us to support in vitro diagnostic medical device commercialization—together with Medicover’s experience in clinical routine testing and presence in the European Union to provide this companion diagnostic to a broader patient population,” Jay Patel, MD, vice president of Clinical Trials and PharmaDX at ARUP, said in a company press release. “Our ultimate goal, in partnership with BioMarin, is to make AAV5 DetectCDx available in all markets where ROCTAVIAN will be authorized so that patients have access to this revolutionary treatment.”
“This collaboration represents a milestone for our precision medicine activities,” said Rainer Metzger, managing director of Integrated Clinical Services at Medicover, which provides diagnostic and healthcare services in Europe. “We have been impressed by the ARUP team and look forward to working with them further.”
Trial data backed use of DetectCDx as a Roctavian companion diagnostic test
AAV5 DetectCDx, the first ARUP-developed test to be performed in Europe, was cleared for use as a Roctavian companion diagnostic test in both the EU and the U.S. based on data from clinical trials, where it was employed to determine patient eligibility to the gene therapy. Last month, the test also was cleared for use under stricter EU rules that will become effective in 2026.
“Our partnership with Medicover demonstrates how patient populations can better be served by a successful collaboration among healthcare providers and industry partners,” said Emily Coonrod, ARUP’s senior director of CDx.
Roctavian uses a modified and harmless version of the AAV5 to deliver to liver cells a shorter, but working copy of F8, the gene that provides instructions to produce the blood clotting factor (FVIII) and that is mutated in people with hemophilia A.
As the liver is the body’s main producer of blood clotting factors, the gene therapy is expected to restore FVIII levels, ultimately mitigating the risk of bleeds, and prospectively lowering or eliminating the need for regular preventive treatment.
“Our PharmaDx group exists to support companion diagnostic testing for the development of cutting-edge therapies in diseases with significant unmet clinical need,” said Patel. “We are honored to serve patients and our partners in this capacity, which has expanded even further through our partnership with Medicover.”
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