Today (December 20), the US FDA announced the approval of Spark Therapeutics' innovative gene therapy Luxturna (voretigeneneparvovec-rzyl) for the treatment of children and adults with hereditary retinopathy (IRD) caused by the biallelic RPE65 mutation. In addition, Luxturna is also the first "direct-dose" gene therapy approved in the United States to target specific gene mutations. Previously, Luxturna had received the FDA's priority review and breakthrough therapy. It is understood that IRD (hereditary retinal disease) is a series of rare blinding diseases caused by one of more than 220 different genes. IRD patients with double allele RPE65 mutations will be due to childhood or early adulthood. Reduced light sensitivity and involuntary nystagmus tremors often cause night blindness. As the disease progresses, the patient's retinal photoreceptor cells (pigment cells) gradually lose function and necrosis, eventually leading to complete loss of visual function, most It affects children or adolescents. At present, there are no effective medical plans and approved drugs to treat the disease. However, the emergence of Luxturna has brought hope for the cure of patients with this disease. Developed by Spark Therapeutics Pharmaceuticals, Inc., Luxturna is a true genetic drug that is expected to cure blind genetic disorders in children at one time. Luxturna uses adeno-associated virus technology to introduce healthy RPE65 gene into retinal cells for expression. Thus, the retinal photoreceptor cells with normal functions may survive, so the patient is also expected to restore visual perception. In a previous phase III clinical trial, Luxturna has achieved good results, and 18 of the 20 testers achieved better results in the maze test after one year of treatment, 13 of them Passing the normal minimum human vision test, more than half of the subjects were able to see three or more lines of characters on the eye chart, and there were no serious adverse events and serious immune events in the trial. However, for Luxturna's approved listing, some industry insiders said that it will face challenges in pricing and reimbursement models, because there is no precedent in the United States for reference, and there are not many other relevant experiences in other places. To date, Glybera (alipogenetiparvovec), the first gene therapy marketed in the European Union for the treatment of lipoprotein lipase deficiency (LPLD), has been withdrawn from the market due to lack of demand, with a cost of up to 1 million euros per treatment. GlaxoSmithKline will also launch its gene therapy Strimvelis for the treatment of adenosine deaminase deficiency combined with immunodeficiency (ADA-SCID), but the company has no plans to discuss the pricing of its therapies. Disposable Nonwoven 3Ply Face Mask Child Disposable Facemask,,Civilian Protection Personal Health Face Mask,Disposable Nonwoven 3Ply Face Mask Dongguan Keyutai Mask Co., Ltd. , https://www.maskkytai.com
Today, the first gene therapy in the US was approved