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BioMarin’s Revolutionary Gene Therapy, Roctavian, Launches in Europe

BioMarin Pharmaceutical has introduced its new gene therapy, Roctavian, in Europe, marking a significant breakthrough for severe haemophilia A patients. The therapy, first administered in Germany, provides a one-time treatment allowing patients’ bodies to produce the essential factor VIII protein for blood clotting autonomously, possibly eliminating frequent infusions currently required. Following its recent approval by the US FDA, the European Commission has granted conditional marketing authorization for Europe. Roctavian offers hope for improved quality of life for haemophilia A patients worldwide.

BioMarin Pharmaceutical Inc., has made a announcement regarding its haemophilia A gene therapy, Roctavian. For the first time in Europe, a patient in Germany with severe haemophilia A has been treated with this one-time infusion therapy.

Haemophilia A is a genetic disorder where sufferers lack enough of the factor VIII protein, which aids in blood clotting. This deficiency can lead to life-threatening bleeds, especially in those with the disease’s most severe form. Presently, those severely affected undergo frequent intravenous infusions to manage the disease, but these aren’t always effective in preventing “breakthrough bleeds” leading to joint damage. Roctavian, enables patients to naturally produce the clotting factor VIII protein.

Roctavian, which secured conditional marketing approval from the European Commission in August 2022, aims to transform treatment paradigms. Administered once, it delivers a functional gene to patients, allowing their bodies to produce the crucial factor VIII protein autonomously, potentially eliminating the need for ongoing haemophilia treatments.

“Today represents an important milestone for the hemophilia community, and for patients and physicians around the world seeking access to Roctavian. We look forward to more people gaining access to Roctavian in the rest of Europe, as well as the USA, where Roctavian recently received FDA approval,” said Jeff Ajer, BioMarin’s executive vice president.

This European milestone follows closely on the heels of Roctavian’s approval by the US Food and Drug Administration, highlighting the rapid international interest in this innovative treatment.

“The burden of severe hemophilia A for people who are living with the condition is substantial, and there remains a significant unmet need for effective treatments that do not require chronic therapy. This one-time infusion represents an important milestone, offering new hope and potential, for eligible individuals in Germany. Bringing this therapy to all those who can benefit is critical to improve outcomes for individuals with severe hemophilia A,” said Dr. Robert Klamroth, chief physician of the Center for Hemophilia and Hemostaseology in Berlin.

Haemophilia A, impacting about 1 in 10,000 individuals, remains a severe health concern. The advent of Roctavian might just signal the dawn of a new era in haemophilia care, potentially improving the quality of life for countless patients.

About Roctavian

Roctavian is an adeno-associated virus vector–based gene therapy indicated for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity < 1 IU/dL) without antibodies to adeno–associated virus serotype 5 (AAV5) detected by an FDA–approved test. The one-time infusion works by delivering a functional gene that is designed to enable the body to produce FVIII on its own, reducing the need for ongoing prophylaxis, demonstrated by results reported through a 3-year study period. The European Commission (EC) granted conditional marketing authorization to Roctavian on August 24, 2022. The reference for the article:>>>

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