Core Viewpoint - The article discusses the rising trend of high-priced gene therapies, particularly focusing on Novartis's newly approved gene therapy Itvisma for spinal muscular atrophy, priced at $2.59 million, which highlights the challenges of commercialization and reimbursement in the gene therapy market [3][6]. Pricing and Market Dynamics - Novartis's Itvisma is priced at $2.59 million, surpassing its earlier product Zolgensma, which was priced at $2.125 million [3][4]. - The global list of the most expensive drugs includes several gene therapies, with the top position held by Kyowa Kirin's Lenmeldy at $4.25 million, followed by CSL's Hemgenix at $3.5 million [4][5]. - The article notes that despite the high prices, many gene therapies face commercialization challenges, as seen with Bluebird Bio's products, which have not achieved significant sales despite their high price tags [5][6]. Sales Performance and Market Challenges - Zolgensma generated $1.2 billion in sales in 2024, maintaining its performance from the previous year, while Bluebird Bio's Lyfgenia only achieved $10 million in sales in Q3 2024 [6][7]. - The article emphasizes the disparity between the high prices of gene therapies and their actual market performance, indicating a potential "value without market" scenario [5][6]. Future Outlook and Market Growth - The global gene therapy market is projected to grow from $9 billion in 2024 to $11.5 billion in 2025, with an expected compound annual growth rate of 27.6% [7]. - The article suggests that a balance between innovation and accessibility is crucial for the future of gene therapies, with a collaborative approach needed from payers, regulators, and pharmaceutical companies [7][8].

2025年迄今，全球最贵药物排名位居榜首的是生物医药公司协和麒麟（Kyowa Kirin）的一款治疗异染 性脑白质营养不良（MLD）的基因疗法Lenmeldy，单剂定价425万美元。这款药物于2024年3月获美国 FDA批准上市，定价打破了此前由CSL公司开发的血友病B基因疗法Hemgenix的纪录。Hemgenix单剂价 格为350万美元，曾"霸榜"全球最昂贵药物头衔长达16个月。该药物于2023年11月获美国FDA批准。 在全球最贵药物排名中，单剂价格超过300万美元总共有四个药，除了Hemgenix之外，美国基因疗法公 司Sarepta Therapeutics的一款治疗杜氏肌营养不良症（DMD）的基因疗法Elevidys单剂售价320万美元， 排名第三；蓝鸟生物的镰状细胞疗法Lyfgenia和另一款治疗脑肾上腺脑白质营养不良（CALD）的药物 Skysona，价格分别为310万美元和300万美元。 近日，一款"一针抵一套豪宅"的罕见病药物引发业内关注。该药物是诺华最新获得美国FDA批准用于治 疗两岁及以上已确诊携带运动神经元存活基因1（SMN1）基因突变的脊髓性肌萎缩症（SMA）患者的 基因疗法Itv ...