Breaking News
March 24, 2018 - Kids with severe brain injuries may develop ADHD: study
March 24, 2018 - Researchers explore ways to help older adults taper off and stop using sedatives
March 24, 2018 - Back pain being mismanaged globally
March 24, 2018 - Fingerprint test accurately and noninvasively detects heroin, cocaine users
March 24, 2018 - Leading experts to promote cardiovascular health at EuroPrevent 2018
March 24, 2018 - A Role for Rituximab in Lupus?
March 24, 2018 - New osteoarthritis genes discovered
March 24, 2018 - Maternal intake of DHA supplement linked to higher fat-free body mass in children
March 24, 2018 - Royal College of Pathologists‘ bulletin provides summary of Tissue Handling Workshop
March 24, 2018 - Maternal alcohol use early in pregnancy may be risk factor for infant abdominal malformation
March 24, 2018 - Savara Initiates Phase 2a Clinical Study of Molgradex for the Treatment of NTM Lung Infection
March 24, 2018 - Accelerated WBI Should be the Norm for Most Breast Cancers
March 24, 2018 - Experts seek to standardize treatments for childhood rheumatic diseases
March 24, 2018 - Foil-based measuring chip rapidly detects Legionella
March 24, 2018 - Bariatric surgery linked to positive outcomes in very obese adolescents with type 2 diabetes
March 24, 2018 - Are there risks from secondhand marijuana smoke? Early science says yes.
March 24, 2018 - New University of Bath project seeks to make injections safer
March 24, 2018 - Higher-dose RT does not improve survival but reduces recurrence risk for prostate cancer patients
March 24, 2018 - Researchers examine link between knee pain and depression in older adults
March 24, 2018 - FDA Alert: BD Vacutainer Blood Collection Tubes by Becton, Dickinson and Company (BD): Class I Recall
March 24, 2018 - Daytime Sleepiness Linked to Amyloid Accumulation Without Dementia
March 24, 2018 - Energy storehouses in the brain may be source of Alzheimer’s, targets of new therapy
March 24, 2018 - Praising people with autism shows promise for producing more exercise
March 24, 2018 - Using harmless red or infrared light to diagnose breast cancer
March 24, 2018 - Clash over abortion hobbles a health bill. Again. Here’s how.
March 23, 2018 - Virtual nature environment could be new way to recover from stress
March 23, 2018 - New study identifies key cellular mechanisms behind vascular aging in mice
March 23, 2018 - Nightmares Common Among U.S. Troops, But Seldom Reported
March 23, 2018 - Another Record Low for Tuberculosis in U.S.
March 23, 2018 - Changes in the eye connected to a decline in memory
March 23, 2018 - Radiologist creates dramatic teaching tool using power of VR
March 23, 2018 - Grilled meat could be raising the risk of hypertension finds study
March 23, 2018 - Mutations found in bassoon gene may help explain cause of rare brain disorder
March 23, 2018 - Childhood Brain Injuries May be Linked to ADHD Years Later
March 23, 2018 - Why treating addiction with medication should be carefully considered
March 23, 2018 - Researchers make key discovery about cellular pathway linked to myriad of diseases
March 23, 2018 - Researchers uncover cause of rare childhood neurodegenerative disease
March 23, 2018 - Measles infection in early childhood could contribute to later COPD
March 23, 2018 - Opioid painkiller is top prescription in 11 states
March 23, 2018 - Sienna Biopharmaceuticals Announces First Patient Dosed In Proof-of-Concept Trial of Topical By Design™ JAK Inhibitor SNA-125 for Atopic Dermatitis
March 23, 2018 - In Teen Girls, Neural Patterns May Drive Emotional Resilience
March 23, 2018 - Gene-based test for urine detects, monitors bladder cancer
March 23, 2018 - BD to introduce new digital solution for IV chemotherapy administration process at EAHP 2018
March 23, 2018 - New computational method helps to identify tumor cell mutations with greater accuracy
March 23, 2018 - Researchers identify potential obesity treatment in freezing hunger-signaling nerve
March 23, 2018 - Wales participates in the 100,000 Genomes Project
March 23, 2018 - 24-Hr Paging Cuts ED Visits for Kids with Endocrine Issues
March 23, 2018 - The brain learns completely differently than we’ve assumed since the 20th century
March 23, 2018 - Less nutritious diet mainly contributes to Type 2 diabetes among U.S.-based South Asians
March 23, 2018 - Stony Brook Medicine expert provides tips for healthy diet to decrease cancer risk
March 23, 2018 - New findings could have revolutionary impact on quality of life of older people
March 23, 2018 - Restoring enzyme may help reverse effects of vascular aging, study shows
March 23, 2018 - Protein profiling reveals new prostate cancer mechanisms
March 23, 2018 - Depression may be linked to increased risk of atrial fibrillation
March 23, 2018 - FDA Takes Aim at Flavored Tobacco
March 23, 2018 - SMART Strategy Lowers Asthma Exacerbation Risk
March 23, 2018 - Cold open water plunge provides instant pain relief
March 23, 2018 - Portable and wearable technology supports future of military medical devices
March 23, 2018 - Patients with vascular malformations have poor health-related quality of life
March 23, 2018 - Researchers develop unique technology to overcome global antibiotic resistance crisis
March 23, 2018 - New DOD grant to support testing of promising therapy for triple-negative breast cancer
March 23, 2018 - Novel vaccine technologies can help better prepare for future infectious disease threats
March 23, 2018 - OncoBreak: Colonoscopy TV; Coverage for Genomic Testing; Care for Caregivers
March 23, 2018 - For some surgeries, nerve blocks mean better outcomes, fewer opioids
March 23, 2018 - Maternal obesity and androgen excess induce sex-specific anxiety in offspring, study suggests
March 23, 2018 - The tale of Theranos and the mysterious fire alarm
March 23, 2018 - USC researchers create algorithm to optimize substance abuse intervention groups
March 23, 2018 - Impulsivity may be associated with greater weight loss during treatment in obese children
March 23, 2018 - CTI BioPharma Announces Publication of Pacritinib Phase 3 PERSIST-2 Clinical Trial in JAMA Oncology
March 23, 2018 - Senate Panel Addresses Native Americans’ Opioid Troubles
March 23, 2018 - Brain connections in schizophrenia
March 23, 2018 - Mental health assessment in health checks can help detect psychologically vulnerable people
March 23, 2018 - New test for urothelial cancers offers less invasive, more accurate detection
March 23, 2018 - Groundbreaking 100,000 Genomes Project achieves important milestone to transform NHS care
March 23, 2018 - Mice getting a new lease of life with anti-aging pills
March 23, 2018 - Obesity kills taste buds and dulls taste sensation finds study
March 23, 2018 - Medical students get less formal education in radiation oncology, study finds
March 23, 2018 - Researchers find investigational compound to treat triple negative breast cancer after brain metastasis
March 23, 2018 - Researchers develop wearable system to monitor electrical activity in the stomach over 24 hours
March 23, 2018 - A Different Opioid Crisis | Medpage Today
FDA Approves Gene Therapy for Rare Form of Blindness: MedlinePlus Health News

FDA Approves Gene Therapy for Rare Form of Blindness: MedlinePlus Health News

image_pdfDownload PDFimage_print

TUESDAY, Dec. 19, 2017 (HealthDay News) — A new gene therapy to treat children and adults with a rare type of inherited vision loss has been approved by the U.S. Food and Drug Administration.

It’s the first gene therapy approved in the United States for a disease caused by mutations in a specific gene, and only the third gene therapy ever approved.

People with this disorder “now have a chance for improved vision, where little hope previously existed,” Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said in an agency news release.

The drug — Luxturna (voretigene neparvovec-rzyl) — can be used to treat people with a condition called biallelic RPE65 mutation-associated retinal dystrophy. It causes vision loss and, in some people, may lead to complete blindness. The condition affects 1,000 to 2,000 people in the United States, according to the FDA.

“One of the best things I’ve ever seen since surgery are the stars. I never knew that they were little dots that twinkled,” therapy recipient Mistie Lovelace said at a public hearing in October. As reported by the Associated Press, Lovelace, from Kentucky, was among several patients urging the FDA to approve the treatment.

Development of the drug began with research started about 25 years ago at the University of Pennsylvania and Children’s Hospital of Philadelphia. Its co-leader was Dr. Jean Bennett, an ophthalmology professor at the university’s Perelman School of Medicine and the Scheie Eye Institute.

“I’ve witnessed the dramatic changes in the vision of patients who would have otherwise lost their sight, and feel exhilarated that this therapy will now make a difference in the lives of more children and adults,” Bennett said in a university news release.

“I’m hopeful that the path we’ve made with this research, with the help of our collaborators near and far, will be useful to other groups, so that other gene therapies can be developed faster and help more people with other diseases,” she said.

The FDA approved the treatment after a Phase 3 study of 31 people measured how their ability to navigate an obstacle course at various light levels changed over a year. Those who took Luxturna showed significant improvements in their ability to complete the obstacle course at low light levels, compared with people who did not take the drug.

The most common problems from treatment with Luxturna were eye redness, cataract, increased intraocular pressure and retinal tear, the FDA noted.

The drug’s maker, Spark Therapeutics, said it will launch a study to assess the long-term safety of Luxturna.

The FDA recommends the drug for those with a confirmed case of the condition who are 1 year and older. It’s a one-time injectable treatment, the researchers said.

Like other gene therapies approved so far, this treatment will not be cheap. Spark said it would release pricing information in January but that its analysis would put the cost of the therapy at about $1 million, according to the AP.

The researchers said that through the clinical trials leading up to the drug’s approval, 41 people have been treated with the drug.

“It has been amazing watching them grow up,” Bennett said. Many are now able to read what’s on their classrooms’ chalkboards, go grocery shopping, recognize people’s faces and get jobs, “among other activities that seemed impossible before,” she added.

The FDA commissioner, Dr. Scott Gottlieb, said in the agency news release that the 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.”

Gottlieb pointed out that “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,” he noted.

“We’re at a turning point when it comes to this novel form of therapy,” he said, adding that the FDA is “focused on establishing the right policy framework to capitalize on this scientific opening.”

Gottlieb said that, in the next year, the FDA would issue “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.”

SOURCES: U.S. Food and Drug Administration, news release, Dec. 19, 2017; Penn Medicine, news release, Dec. 19, 2017; Associated Press

News stories are written and provided by HealthDay and do not reflect federal policy, the views of MedlinePlus, the National Library of Medicine, the National Institutes of Health, or the U.S. Department of Health and Human Services.

Tagged with:

About author

Related Articles