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www.npr.org/sections/health-shots/2020/07/20/800556057/gene-therapy-shows...

The first gene therapy for hemophilia could be approved by the FDA within six months, according to the drugmaker, raising hopes among families. But the drug's price could be $3 million per patient.

www.inquirer.com/health/oxbryta-adakveo-approved-fda-sickle-cell-disease...

The new drugs come as dozens of experimental therapies are in development, including some genetically engineered treatments that may offer a cure by repairing hemoglobin’s ability to carry oxygen. Early this year, for example, the FDA gave “fast track designation” to Vertex Pharmaceuticals’ gene-edited stem cell therapy for the disease.

www.drugs.com/history/roctavian.html

Roctavian FDA Approval Status. FDA Approved: No Brand name: Roctavian Generic name: valoctocogene roxaparvovec Company: BioMarin Pharmaceutical Inc. Treatment for: Hemophilia A Roctavian (valoctocogene roxaparvovec) is an investigational gene therapy in development for the treatment of hemophilia A.

www.biopharma-reporter.com/Article/2020/05/21/Novartis-gene-therapy-approved...

In March , Novartis was able to announce that Zolegensma (onasemnogene abeparvovec) that it had received a positive opinion on its application for marketing approval by the European Medicines Agency.. Yesterday this was followed up by the announcement that the European Commission (EC) had provided the gene therapy conditional approval for the treatment of patients with spinal muscular atrophy.

www.the-scientist.com/news-opinion/fda-approves-gene-therapy-for-spinal...

The drug’s price tag, he says, drains resources from society, and it’s not alone. The first gene therapy, approved in 2017 and designed to treat a genetic condition that causes blindness, costs $425,000 for each eye.

www.scientificamerican.com/article/fda-plans-to-speed-path-to-approval-for...

The Food and Drug Administration will soon be alerting companies that certain gene therapies in development can qualify for less arduous review at the agency, Commissioner Scott Gottlieb said Tuesday.

cadth.ca/dv/ieht/gene-therapy-overview-approved-and-pipeline-technologies

According to the FDA, gene therapy is “the administration of genetic material to modify or manipulate the expression of a gene product or to alter the biological properties of living cells for therapeutic use.” 3 In Canada, gene therapies are included in the definition of “drug” under the Food and Drugs Act and are regulated under the ...

www.cnn.com/2017/12/20/health/fda-gene-therapy-blindness-bn

The FDA has approved Luxtruna for patients with a rare inherited eye disease. It's the third gene therapy to win US approval, the first for a genetic disease.

payorsolutions.cvshealth.com/insights/gene-therapies-promising-costly-complex

The FDA recently approved Luxturna, a type of vector-based gene therapy that is injected directly into a patient’s retina (back of the eye) to help reverse vision loss in pediatric and adult patients with a specific type of progressive blindness due to a mutated gene (RPE65). Vector-based gene therapies are also in development for the ...

www.labiotech.eu/in-depth/atmp-cell-gene-therapy-ema

There is a lot of talk about how cell and gene therapy are going to change medicine, so why have only a few of them reached the market? Over the last decade, only 15 advanced therapy medicinal products (ATMPs) – that is, cell and gene therapies – have received approval in Europe. With four withdrawals, only 11 of them still have a valid marketing authorization.