The guidance documents are intended “to serve as the building blocks of a modern, comprehensive framework for how we’ll help advance the field of gene therapy, while making sure new products meet the FDA’s gold standard for safety and effectiveness,” ​said Gottlieb in a statement​. WCG Spark Therapeutics ( FDA ) has announced recommendations for supporting gene therapies aimed at tacking haemophilia, retinal disorders and rare diseases. It said the suggestions reflecting increasing work in gene therapy could be used by sponsors to speed a drug’s official FDA approval. The FDA has already approved the Gilead Sciences therapy from Novartis , FDA ’ Yescarta and the Luxturna therapy from Spark Therapeutics and Kymriah has now won a second approval. Spark Therapeutics commissioner Scott Gottlieb said the recommendations were intended as building blocks for a comprehensive framework for gene therapy.
Spark Therapeutics ( FDA ) has announced recommendations for supporting gene therapies aimed at tacking haemophilia, retinal disorders and rare diseases. It said the suggestions reflecting increasing work in gene therapy could be used by sponsors to speed a drug’s official FDA approval. The FDA has already approved the Gilead Sciences therapy from Novartis , FDA ’ Yescarta and the Luxturna therapy from Spark Therapeutics and Kymriah has now won a second approval. Spark Therapeutics commissioner Scott Gottlieb said the recommendations were intended as building blocks for a comprehensive framework for gene therapy.