Breaking News
August 17, 2018 - Bacterial activity in child’s mouth may serve as biomarkers for autism spectrum disorder
August 17, 2018 - Scripps Research scientists uncover new approach for treating thrombocytopenia
August 17, 2018 - Mathematical model shows the influence of human behavior on spread of infectious diseases
August 17, 2018 - Valley Hospital achieves Magnet recognition for fourth consecutive time
August 17, 2018 - Researchers describe link between poor oocyte development and oxidative stress in obese mice
August 17, 2018 - Hospitals battle for control over fast-growing heart-valve procedure
August 17, 2018 - AHA: Home-Delivered Meals Keep Heart Failure Patients Out of Hospital
August 17, 2018 - In Southern Mozambique, only half of people diagnosed with HIV enroll in medical care
August 17, 2018 - Researchers discuss techniques to help combat growing epidemic of obesity
August 17, 2018 - Researchers develop novel statistical method to evaluate gene-to-gene interactions linked with cancer
August 17, 2018 - Island Fertility joins Stony Brook Community Medical to provide comprehensive fertility care
August 17, 2018 - Study shows link between thinning of the retina and early sign of Parkinson’s disease
August 17, 2018 - Digital birth control app gets FDA nod
August 17, 2018 - FDA grants approval for first generic version of epinephrine auto-injector
August 17, 2018 - Federal advisory group publishes recommendations on prevention of acute, chronic pain
August 17, 2018 - 3D-printed human body parts to be used as teaching aids for surgical training
August 17, 2018 - U.S. murder, suicide rates climbing again
August 17, 2018 - This is your brain on… roller coasters?
August 17, 2018 - Report discusses whether all newborns should undergo genetic sequencing
August 17, 2018 - UCR receives 2018 Inspiring Programs in STEM Award from INSIGHT Into Diversity magazine
August 17, 2018 - Researchers publish new paper on developing vaccine candidates for Helminthic parasites
August 17, 2018 - Researchers develop new method to diagnose broad range of cancers using malaria protein
August 17, 2018 - Female mosquitoes quickly evolve selective mating behavior when faced with threats
August 17, 2018 - FDA Grants Breakthrough Therapy Designation to Daiichi Sankyo’s FLT3 Inhibitor Quizartinib for Relapsed/Refractory FLT3-ITD AML
August 17, 2018 - Resistance training and exercise motivation go hand-in-hand
August 17, 2018 - A lesson for future doctors: Listen to and learn from your patients
August 17, 2018 - NUS study discovers a bidirectional regulator and shines light on A-to-I RNA editing in cancer cells
August 17, 2018 - Research shows link between high blood levels of omega-3s and better brain function in children
August 17, 2018 - Researchers propose new theory for how rare gene mutations cause Alzheimer’s disease
August 17, 2018 - New project to combat DMD-related fibrosis receives major funding boost
August 17, 2018 - Digital psychiatric therapy can ‘rewire’ the brain in children with ADHD, study shows
August 17, 2018 - Psychologist to assess how the brain maintains precise short-term and long-term memories
August 17, 2018 - Eating white button mushrooms could improve regulation of glucose in the liver
August 17, 2018 - Scientists identify mutational signatures in ovarian cancer
August 17, 2018 - Sun Pharma receives U.S. FDA approval for CEQUA to treat patients with dry eye disease
August 17, 2018 - Teva Announces Updated Indication and Vial Presentation for Granix (tbo-filgrastim) Injection in United States
August 17, 2018 - Study shows DNA methylation related to liver disease among obese patients
August 17, 2018 - Life on the border: Back at Stanford, ready to pitch in
August 17, 2018 - New device for accurately placing hemodialysis catheters on kidney patients
August 17, 2018 - New strategy accelerates, automates process of prototype molecule optimization
August 17, 2018 - Study finds role of autoimmunity in development of COPD
August 17, 2018 - Researchers transform research tool to study neuronal function
August 17, 2018 - Cognitive impairment does not equate to unhappiness in older adults
August 17, 2018 - Peer Comparisons Can Decrease Risky Prescribing Patterns
August 17, 2018 - Susceptible genes identified for childhood chronic kidney disease
August 17, 2018 - Research uncovers miscarriage cause, identifies potential targets for treatment
August 17, 2018 - Bacterial armor could be new target for antibiotics | News Center
August 17, 2018 - FDA expands approval of Vertex’ cystic fibrosis medicine to treat children aged 12 to
August 17, 2018 - Give Your Child a Head Start With Math
August 17, 2018 - Ground-breaking study tests whether rejected livers can be made viable for transplantation
August 16, 2018 - New algorithm could improve diagnosis of rare diseases | News Center
August 16, 2018 - SCHILLER introduces latest generation of ECG device, CARDIOVIT AT-102 G2
August 16, 2018 - Proper treatment, refraining from smoking can reduce heart disease risk from type 2 diabetes
August 16, 2018 - Mount Sinai study could transform treatment for patients with retinal degenerative diseases
August 16, 2018 - Penn researchers develop first mouse model of idiopathic pulmonary fibrosis
August 16, 2018 - Four tips to help prevent fall allergy symptoms
August 16, 2018 - Women’s Preventive Services Initiative says screen all women annually for urinary incontinence
August 16, 2018 - At Stanford, patient discovers the source of her headaches, nausea | News Center
August 16, 2018 - To Prevent Injuries in Young Baseball Players, Chris Ahmad Reaches Out to Parents
August 16, 2018 - Restoring blood flow may be linked to longer survival in patients with critical limb ischemia
August 16, 2018 - New model of genetically engineered immune cells may help fight solid tumors
August 16, 2018 - Maternal stress increases anxious and depressive-like behaviors in female offspring
August 16, 2018 - Childhood exposure to secondhand smoke increases risk of COPD death in adulthood
August 16, 2018 - Scientists uncover key control mechanism of DNA replication
August 16, 2018 - NIH begins first-in-human trial of experimental live, attenuated Zika virus vaccine
August 16, 2018 - Two diabetes medications don’t slow progression of type 2 diabetes in youth
August 16, 2018 - 5 Questions: How Stanford research is making MRI scans safer for kids | News Center
August 16, 2018 - Columbia Celebrates 25th Anniversary of White Coat Ceremony
August 16, 2018 - Phonak’s new smallest and most discreet Virto B-Titanium hearing aid
August 16, 2018 - New project aims to study growth of water-based microorganisms
August 16, 2018 - Immune cell found to play important role in photosensitivity
August 16, 2018 - Higher social dominance linked to faster decision-making in men
August 16, 2018 - Blood test in early pregnancy could determine a woman’s later risk for gestational diabetes
August 16, 2018 - New research confirms link between DDT exposure and autism
August 16, 2018 - Neurodevelopmental Anomalies, Birth Defects Linked to Zika ID’d
August 16, 2018 - Risk of heart failure up in ALVSD patients with diabetes
August 16, 2018 - Exercise reduces symptoms and fatigue in patients with chronic kidney disease
August 16, 2018 - Study reveals role of RUNX proteins in DNA repair
August 16, 2018 - New research finds no harm from average salt consumption
August 16, 2018 - Researchers develop new way of testing bacterial resistance to antibiotics
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