21st December 2017 First gene therapy for an inherited condition approved in the U.S. The FDA has approved Luxturna – the first gene therapy for an inherited condition in the U.S. – for patients with a form of vision loss.
Gene therapy is a new and emerging form of medicine that seeks to treat or prevent diseases by augmenting, replacing or suppressing mutated genes with functional copies. It addresses the root cause of an inherited disease, by enabling the body to produce proteins necessary to restore health or to stop making a harmful protein or proteins, with the potential of bringing back function in diseased cells. To deliver the functional gene into the cell, a vector is used to transport the desired gene and is delivered either intravenously or injected into specific tissue. The goal is to enable, through a one-time administration of gene therapy, lasting therapeutic effects. It is now known that over 80% of rare diseases are based on genetic mutations or are genetic conditions. When all of the different potential inherited conditions are put together, it accounts for over 7,000 rare diseases that affect 30 million people in the U.S. alone. This week, the U.S. Food and Drug Administration (FDA) approved Luxturna (voretigene neparvovec), a new gene therapy, to treat children and adults with an inherited form of vision loss that may result in blindness. "Today's approval marks another first in the field of gene therapy – both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss," said FDA Commissioner Scott Gottlieb, M.D. "This milestone reinforces the potential of this approach in treating a wide range of challenging diseases. The culmination of decades of research has resulted in three gene therapy approvals this year for patients with serious and rare diseases." In August, the FDA approved Kymriah, the first gene therapy for leukaemia. A second gene-based treatment, called Yescarta, was approved in October for a form of lymphoma. Luxturna is the first gene therapy approved in the U.S. for an inherited condition. "I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses," Gottlieb added. "We're at a turning point when it comes to this novel form of therapy. Next year, we'll begin issuing a suite of disease-specific guidance documents on the development of specific gene therapy products to lay out modern and more efficient parameters – including new clinical measures – for the evaluation and review of gene therapy for different high-priority diseases where the platform is being targeted."
Luxturna is approved for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy, which leads to vision loss and may cause complete blindness in certain people. Hereditary retinal dystrophies are a broad group of genetic retinal disorders associated with progressive visual dysfunction and are caused by mutations in any one of more than 220 different genes. Biallelic RPE65 mutation-associated retinal dystrophy affects between 1,000 and 2,000 patients in the U.S. Luxturna works by delivering a normal copy of the RPE65 gene directly to retinal cells, via a subretinal injection. These retinal cells then produce the normal protein that converts light to an electrical signal in the retina to restore vision loss. Luxturna uses a naturally occurring adeno-associated virus, which has been modified using recombinant DNA techniques, as a vehicle to deliver the normal RPE65 gene to retinal cells. "The approval of Luxturna further opens the door to the potential of gene therapies," said Peter Marks, Ph.D., the director of the FDA's Center for Biologics Evaluation and Research (CBER). "Patients with biallelic RPE65 mutation-associated retinal dystrophy now have a chance for improved vision, where little hope previously existed." Luxturna will be manufactured by Spark Therapeutics at their manufacturing facility in West Philadelphia. The gene therapy will be administered at selected treatment centres in the U.S. by leading retinal surgeons, who will receive surgical training provided by Spark Therapeutics on the administration procedure. Luxturna is expected to be available in these treatment centres during the first quarter of 2018.
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