Breaking News
May 27, 2018 - Study reveals striking disparities in health care access and quality across most nations
May 27, 2018 - The Yogi masters were right—meditation and breathing exercises can sharpen your mind
May 27, 2018 - SLU researcher aims to find solutions for diabetes patients at risk of hypoglycemia
May 27, 2018 - Scientists uncover the cause of insulin resistance in obesity
May 27, 2018 - $2.3 million NIH grant to support new project on oxytocin neurons and social behavior
May 27, 2018 - Less Driving Tied to Lower Cardiovascular Disease Risk
May 27, 2018 - Genetics Home Reference: LMNA-related congenital muscular dystrophy
May 27, 2018 - Long-term psychological study confirms time is the best medicine against homesickness
May 27, 2018 - Study explores if CPAP treatment can improve sexual QOL for sleep apnea patients
May 27, 2018 - Study investigates role played by brain in prosocial behavior
May 27, 2018 - New Guidelines Mean 1 in 3 Adults May Need Blood Pressure Meds
May 27, 2018 - Cerebrospinal Fluid (CSF) Analysis: MedlinePlus Lab Test Information
May 27, 2018 - Kids in tough neighborhoods head to ER more often
May 27, 2018 - Exercise alters brain’s dopamine system to help treat addiction, study finds
May 27, 2018 - Sepsis patients treated and released from ED for outpatient follow-up experience good outcomes
May 27, 2018 - Initiative cuts overuse of tests, treatments for bronchiolitis
May 27, 2018 - Study links ‘sleep spindles’ to memory reactivation
May 27, 2018 - Scientists develop new method to speed up genome evolution of baker’s yeast
May 27, 2018 - Sunscreen pills are fake says FDA
May 27, 2018 - Study finds increasing wealth gap between households of seniors and families with children
May 27, 2018 - Link between tuberculosis and Parkinson’s disease discovered
May 27, 2018 - Doctors call on health authorities for permission to provide stroke patients with life-saving treatment
May 26, 2018 - Couples who eat seafood-rich diet tend to get pregnant faster
May 26, 2018 - NIH summit presents recommendations to accelerate treatment development for Alzheimer’s disease
May 26, 2018 - Medication-related harm found to be common among older adults, but preventable
May 26, 2018 - Lunaphore and Vitro announce partnership to develop ISH protocols for RNA, DNA targets
May 26, 2018 - Cryoablation Efficacious for Cancer Pain, Review Finds
May 26, 2018 - Link between IBD and Parkinson’s might allow doctors to slow down condition
May 26, 2018 - Study finds fewer than 5% of low-income, urban mothers use prenatal vitamins before pregnancy
May 26, 2018 - California hospitals urge moms to favor breast milk over formula
May 26, 2018 - Most concussion patients do not receive follow-up care after hospital discharge, says study
May 26, 2018 - Lifetime risks of developing Alzheimer’s dementia vary by age, gender
May 26, 2018 - Researchers find novel ways to improve participation in clinical research
May 26, 2018 - Researchers develop methods for measuring free-base nicotine levels in e-cigarettes
May 26, 2018 - AHA: Preterm Birth Could Warn of Mom’s Future Heart Risks
May 26, 2018 - Some calories more harmful than others
May 26, 2018 - Study links cell size with commitment to division
May 26, 2018 - Researchers develop new, rapid blood test to detect liver damage
May 26, 2018 - Researchers discover cascade of immune processes linked to poor outcomes in aggressive breast cancer
May 26, 2018 - New research will use mathematics to solve mysteries in cell biology
May 26, 2018 - Proposed National Resilience Strategy to reverse catastrophic increases in ‘deaths of despair’
May 26, 2018 - Mice remain slim on burger diet
May 26, 2018 - BMC receives $13.5 million award to test methods for delivering childhood anxiety treatment
May 26, 2018 - ‘Right to Try Act’ will not benefit terminally-ill patients
May 26, 2018 - Study reveals novel statistical algorithm to identify potential disease genes
May 26, 2018 - Two genes play vital roles in malignant brain cancer
May 26, 2018 - Study explores link between groundwater lithium and diagnoses of bipolar disorder, dementia
May 26, 2018 - Researchers reveal stimulatory effects of myelin on young neural cells
May 26, 2018 - Small part of cellular protein that helps form long-term memories also drives neurodegeneration
May 26, 2018 - Four-legged friends can have heart issues, too
May 26, 2018 - Scientists create small, self-contained spaces inside mammalian cells
May 26, 2018 - Better Social Support Network Protects Black Men Against HIV
May 26, 2018 - National Heart, Lung, and Blood Institute (NHLBI)
May 26, 2018 - Burnout, depression can affect ophthalmology residents, study finds
May 26, 2018 - Latinos and African Americans more likely to experience serious depression than Whites
May 26, 2018 - Data from past epidemic could help improve response to future Ebola outbreaks
May 26, 2018 - Researchers provide insight into how the memory molecule limits brain plasticity
May 26, 2018 - OSU biologist describes ‘restoration ecology’ approach toward patient health
May 26, 2018 - New approach to study brown fat could aid in finding treatments for obesity
May 26, 2018 - UCI Center on Stress & Health receives NIH funding to develop digital health interventions
May 26, 2018 - Could More Fish in the Diet Boost Sex Lives and Fertility?
May 26, 2018 - NTU Singapore and SERI invent new scope to diagnose glaucoma
May 26, 2018 - Cancer cells co-opt pain-sensing ‘neural channel’ to increase tolerance against oxidative stress
May 26, 2018 - Study uncovers why pesticide exposure increases Parkinson’s disease risk in some people
May 26, 2018 - Study finds link between lead exposure and fertility rates
May 26, 2018 - Causes and treatment of acute heart failure vary by region, registry shows
May 26, 2018 - Delivery of standardized diabetes care could help achieve equitable health outcomes for all patients
May 26, 2018 - FDA authorizes marketing of OsteoDetect software for detecting wrist fractures
May 26, 2018 - HSE experts suggest new way of looking at infantilism
May 26, 2018 - Children and adolescents growing up in extreme societal conditions more likely to resort to violence
May 26, 2018 - New study puts forth most comprehensive tree of life for malaria parasites
May 26, 2018 - UVA researchers establish new guidelines for explorers of the submicroscopic world inside us
May 26, 2018 - Princeton Instruments and C-SOPS announce collaboration on innovative pharmaceutical technology
May 26, 2018 - New research shows why babies need to move in the womb
May 26, 2018 - UK steps forward to tackle global antimicrobial resistance
May 26, 2018 - CRISPR-Cas9-based strategy allows researchers to precisely alter hundreds of different genes
May 26, 2018 - Novoheart announces next generation of ‘Human heart-in-a-jar’ technology for advanced drug discovery
May 26, 2018 - UT Southwestern-led researchers find new way to determine prognosis of invasive kidney cancer
May 26, 2018 - Researchers develop film to prevent bacteria from growing on dental retainers and aligners
May 26, 2018 - Mobile health intervention for people with serious mental illness as effective as clinic-based treatment
FDA approves new gene therapy targeting rare form of inherited vision loss

FDA approves new gene therapy targeting rare form of inherited vision loss

image_pdfDownload PDFimage_print

The U.S. Food and Drug Administration today approved Luxturna (voretigene neparvovec-rzyl), a new gene therapy, to treat children and adult patients with an inherited form of vision loss that may result in blindness. Luxturna is the first directly administered gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene.

“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 -; and this milestone reinforces the potential of this breakthrough 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. I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses,” said FDA Commissioner Scott Gottlieb, M.D. “We’re at a turning point when it comes to this novel form of therapy and at the FDA, we’re focused on establishing the right policy framework to capitalize on this scientific opening. 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 that leads to vision loss and may cause complete blindness in certain patients.

Hereditary retinal dystrophies are a broad group of genetic retinal disorders that are 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 approximately 1,000 to 2,000 patients in the U.S. Biallelic mutation carriers have a mutation (not necessarily the same mutation) in both copies of a particular gene (a paternal and a maternal mutation). The RPE65 gene provides instructions for making an enzyme (a protein that facilitates chemical reactions) that is essential for normal vision. Mutations in the RPE65 gene lead to reduced or absent levels of RPE65 activity, blocking the visual cycle and resulting in impaired vision. Individuals with biallelic RPE65 mutation-associated retinal dystrophy experience progressive deterioration of vision over time. This loss of vision, often during childhood or adolescence, ultimately progresses to complete blindness.

Luxturna works by delivering a normal copy of the RPE65 gene directly to retinal cells. These retinal cells then produce the normal protein that converts light to an electrical signal in the retina to restore patient’s 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 human RPE65 gene to the retinal cells to restore vision.

“The approval of Luxturna further opens the door to the potential of gene therapies,” said Peter Marks, M.D., Ph.D., 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 should be given only to patients who have viable retinal cells as determined by the treating physician(s). Treatment with Luxturna must be done separately in each eye on separate days, with at least six days between surgical procedures. It is administered via subretinal injection by a surgeon experienced in performing intraocular surgery. Patients should be treated with a short course of oral prednisone to limit the potential immune reaction to Luxturna.

The safety and efficacy of Luxturna were established in a clinical development program with a total of 41 patients between the ages of 4 and 44 years. All participants had confirmed biallelic RPE65 mutations. The primary evidence of efficacy of Luxturna was based on a Phase 3 study with 31 participants by measuring the change from baseline to one year in a subject’s ability to navigate an obstacle course at various light levels. The group of patients that received Luxturna demonstrated significant improvements in their ability to complete the obstacle course at low light levels as compared to the control group.

The most common adverse reactions from treatment with Luxturna included eye redness (conjunctival hyperemia), cataract, increased intraocular pressure and retinal tear.

The FDA granted this application Priority Review and Breakthrough Therapy designations. Luxturna also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The sponsor is receiving a Rare Pediatric Disease Priority Review Voucher under a program intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. A voucher can be redeemed by a sponsor at a later date to receive Priority Review of a subsequent marketing application for a different product. This is the 13th rare pediatric disease priority review voucher issued by the FDA since the program began.

To further evaluate the long-term safety, the manufacturer plans to conduct a post-marketing observational study involving patients treated with Luxturna.

The FDA granted approval of Luxturna to Spark Therapeutics Inc.

Source:

https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm589467.htm

Tagged with:

About author

Related Articles