FDA approved: the first hemophilia A gene therapy authorized for the United States
Roctavian, a new life-changing treatment for people with hemophilia A developed at University College London (UCL; London, UK) and licensed by UCLB to BioMarin Pharmaceuticals Inc. (CA, USA), has been approved by the US FDA and may now be presented as a treatment option for patients with the illness.
Hemophilia A is a rare hereditary bleeding disorder caused by a genetic mutation, which results in a deficiency of the blood clotting protein, factor VIII. Factor VIII deficiency can cause continuous bleeding due to the lack of clotting after an injury and can be life-threatening in the most serious of cases. People with hemophilia A currently require continuous treatment throughout their lives, with frequent injections required to replace the absent factor VIII.
Roctavian (valoctocogene roxaparvovec) is a revolutionary treatment for hemophilia A, since it is a one-time, long-term treatment, that uses gene therapy to increase patient levels of factor VIII. The active factor VIII gene is supplied through an adeno-associated virus vector, which delivers the necessary genetic material into cells. The gene will then become expressed in the liver, which encourages the production of the factor VIII protein and corrects the ability of the patient’s body to regulate bleeding without regular injections.
Roctavian was developed by Professor Amit Nathwani at the UCL Cancer Institute and Institute of Immunity & Transplantation, his research team at UCL and the team from St. Jude Children’s Research Hospital (TN, USA).
Professor Nathwani commented on this:
“It is humbling to be involved in the creation of the first gene therapy for patients with severe hemophilia A. Current treatment is effective but highly demanding requiring regular lifelong injections. Most severe hemophilia A patients suffer recurrent breakthrough bleeding into joints despite treatment that ultimately leads to disability and chronic pain. The approval of Roctavian is an important and long-awaited advance that addresses the genetic cause of the condition. Roctavian is a one-time gene therapy that offers patients long-term protection from bleeds and avoids the burden of regular infusions.”
Biomarin’s GENEr-8, which has been the longest global Phase III study to date for any gene therapy for hemophilia, was imperative to reaching the FDA approval of Roctavian. The study determined that the treatment reduced the rate of bleeding in a group of 134 individuals for at least 3 years.
Professor Geraint Rees, UCL Vice-Provost for Research, Innovation and Global Engagement, explained:
“Creating the first long-term treatment for severe hemophilia A will change lives, and proving its effectiveness and achieving FDA approval is a major achievement. It is a prime example of how our approach to industry-academic engagement is directly benefitting society.”
UCL is widely regarded as a pioneer in transformational gene treatments. Roctavian’s success follows a string of recent achievements for UCLB’s gene therapy spinouts, including the first patient dosed with a novel gene therapy candidate for Gaucher disease and the NHS’s use of Orchard Therapeutic’s Libmeldy to treat children with metachromatic leukodystrophy, a disease that is otherwise fatal.
Richard Fagan, Director of BioPharm at UCLB, stated:
“Our partnership with BioMarin has spanned over a decade. Since licensing the technology to BioMarin, the company has taken forward Roctavian’s pioneering clinical development, and undertaken some of the most extensive clinical trials for a gene therapy in the world. The successful FDA approval is a significant achievement for UCL and UCLB and ultimately hemophilia patients.”
Sources: FDA press release, www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-adults-severe-hemophilia. Technology Networks press release, www.technologynetworks.com/biopharma/news/first-gene-therapy-for-people-with-haemophilia-a-approved-in-the-us-375795
