第一财经·2025-12-02 12:57Core 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].