(歐盟核准GSK基因療法Strimvelis ) BigPharma 進入新世代: 基因治療?!

葛蘭素史克捷報 基因療法藥物獲准上市 2016-05-28 21:20 中央社 倫敦27日綜合外電報導 全球製藥鉅子葛蘭素史克藥廠（GSK）的基因療法藥物，今天獲得主管機關批准通過，得以上市。根據英國廣播公司（BBC）報導，這款基因療法藥物Strimvelis，用於治療ADA嚴重複合型免疫不全症（ADA-SCID），罹患這種免疫疾病的嬰兒無力對抗日常的感染。這是葛蘭素史克藥廠首款獲准通過的基因療法藥物。評論家表示，這項發展標誌著大藥廠越來越多基因藥物的開端。這種免疫缺乏疾病極為罕見，每年約有24名嬰兒罹患這種疾病。基因療法過關也為開發地中海型貧血和鐮形細胞貧血等較普遍疾病的治療方式鋪路。囊腫纖維症、肌肉萎縮症和多種視覺障礙等，數以百計的遺傳性疾病，肇因於基因的缺陷。基因療法的目的，是透過複製健康的基因到病患身上，治療疾病。基因療法最大的優點，就是只須接受一次性治療，就可能一勞永逸治癒疾病。葛蘭素史克雖然還沒公布這款新治療方式將如何訂價的細節，但研發部門總裁瓦朗斯（Patrick Vallance）表示，公司的目標是讓Strimvelis，以及該公司開發出的更多基因療法，造福更多病患。

GlaxoSmithKline's gene therapy Strimvelis approved in Europe for ADA-SCID

May 27th, 2016/ By: Joe Barber/ GlaxoSmithKline, together with Fondazione Telethon and Ospedale San Raffaele, announced that the European Commission has granted marketing authorisation to the gene therapy Strimvelis for the treatment of children with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). The drugmaker noted that the therapy, which consists of autologous CD34+ cells transduced to express ADA, is the first corrective gene therapy for children to receive regulatory approval anywhere globally. Martin Andrews, head of GlaxoSmithKline's rare disease unit, remarked "this is the start of a new chapter in the treatment of rare genetic diseases and we hope that this therapeutic approach could also be used to help patients with other rare diseases in the future."  Approval of Strimvelis was supported by clinical data collected for 18 children treated with the therapy. All 12 children who participated in the pivotal study were alive three years post-treatment, while all treated patients whose data supported the regulatory submission are alive at present after a median follow-up of seven years.  GlaxoSmithKline, Fondazione Telethon and Ospedale San Raffaele initially submitted Strimvelis, which was previously known as GSK2696273, for European approval for the treatment of ADA-SCID in May last year. The European Medicines Agency's Committee for Medicinal Products for Human Use issued a positive opinion last month backing approval of the therapy.  GlaxoSmithKline has not provided details on the price of Strimvelis, although a person close to the company said last month that the price of the therapy would be "very significantly less than $1 million. GlaxoSmithKline R&D chief Patrick Vallance said the drugmaker will aim to make Strimvelis and other gene therapies as accessible as possible to patients. "We are absolutely committed to getting the price right," Vallance stated, adding "it's obvious that you can't charge a price that is unaffordable."  Gene therapy Glybera (alipogene tiparvovec) gained marketing authorisation in Europe in 2012, for use in patients with lipoprotein lipase deficiency who experience recurring acute pancreatitis. The therapy was later launched at a cost of 1.1 million euros ($1.2 million).