Fidanacogene elaparvovec

Roche Pharma CEO Teresa Graham spoke to Fierce Biotech on the sidelines of the company\'s first-half earnings event.\n The uncertainty around the future of Elevidys hasn’t destroyed Roche’s belief in the potential of gene therapies, the Swiss company’s head of pharma has told Fierce Biotech, as the pharma continues to expand its research reach outside of its traditional oncology focus.Roche and its partner Sarepta Therapeutics both paused trials of Elevidys back in April following the death of a 16-year-old patient in the U.S. Since then, Roche—which markets the drug outside of the U.S.—has stopped all ex-U.S. distribution of the drug in the commercial setting for non-ambulatory patients, while the FDA requested that Sarepta halt Elevidys shipments in the U.S. Earlier this week, Roche paused shipments in all patients in countries where Elevidys’ approvals referenced the FDA.But Roche Pharma CEO Teresa Graham denied Fierce’s suggestion that the issues around Elevidys have altered how the company views gene therapy as a modality.“It doesn’t, actually,” Graham told Fierce in an interview at Roche’s headquarters in Basel, Switzerland on Thursday.“The reality is that gene therapy will be an important modality in the treatment of human health,” Graham continued. “There is a lot of promise in gene therapy. I think what the entire industry is learning is that, scientifically, it\'s just a little harder than we might have thought.” Even before the latest difficulties with Elevidys, Roche had announced a “fundamental reorganization” of Spark Therapeutics, the gene therapy unit the Swiss pharma bought for $4.3 billion in 2019. That overhaul closely followed Roche’s decision to end work on a hemophilia A gene therapy candidate, which was a focus of the Spark acquisition.Roche’s move came against a backdrop of wider industry retreat from gene therapy, with Pfizer pulling its FDA-approved hemophilia B treatment Beqvez. Bluebird bio, a gene therapy pioneer once valued at $10 billion at its peak, was sold for just $49 million upfront to private equity firms.But Roche still sees potential in the modality, according to Graham.“I am firmly convicted that over time we will crack gene therapy and we will be able to deliver really meaningful treatments to patients,” she said.Graham spoke to Fierce on the sidelines of the company’s first-half earnings event, where Roche CEO Thomas Schinecker reminded journalists that 760 boys have successfully received Elevidys in the ambulatory setting.“I\'m really hopeful that we can find a path forward for this medicine,” Graham said in the interview on Thursday. “We clearly need to talk to regulators; we clearly need to have some good conversations about the safety—but we do firmly believe in the risk-benefit of this product.”Not all regulators sound ready to be convinced, with the European Medicines Agency (EMA) announcing Friday morning that it won\'t recommend Elevidys for approval in the ambulatory setting. Roche said in a statement that it “plans to continue to work with the EMA to explore a potential path forward.” Mixed results from emerging pipeline Another drug that Roche is keeping faith in is the Parkinson’s disease prospect prasinezumab. The future of the Prothena Biosciences–partnered asset looked in doubt after the anti-alpha-synuclein antibody failed a phase 2 study in December 2024.But Roche is giving prasinezumab a second chance, with plans to launch a phase 3 trial by the end of the year off the back of an analysis of data from the failed study. Specifically, the pharma pointed to longer-term follow-up data from an open-label portion of the study suggesting a clinical benefit on top of symptomatic treatment in early-stage Parkinson’s.“Given the dramatic unmet need in Parkinson\'s, it does feel like it would be a miss not to explore this,” Graham explained.“There\'s only really been minor improvements in treatments for these patients over the last 50 years, and, in every conversation I\'ve had with [key opinion leaders], they have really encouraged us to take this forward,” she said.“There\'s a little bit of a finger feel that there\'s something there, and we\'re only really ever going to be able to pull the signal out in a phase 3,” Graham added. Roche had less faith in CT-173, an early-stage obesity drug picked up as part of its $2.7 billion acquisition of Carmot Therapeutics in 2023. The Swiss pharma had previously touted the long-acting PYY analog’s potential to drive weight loss past the GLP-1 plateau, but Graham announced at the earnings event that Roche would no longer take CT-173 into human trials after assessing its “developability and competitiveness.”Despite this early-stage setback, Graham still feels that combinations offer Roche a path to carving out its own corner of the increasingly crowded obesity space.“Obesity is inherent in something like 200 other diseases,” she said. “So the ability to actually look at where we believe our portfolio in combination with other drugs can really help address some of these comorbidities is one of the more exciting pieces.”“This is an incredibly big market,” Graham added. “It is very diverse. There are a lot of scientific pathways to explore here.”One of those pathways is amylin, a hormone involved in the regulation of food intake. In March, Roche paid Zealand Pharma $1.6 billion for the long-acting amylin analog petrelintide. Graham sees the drug’s potential growing as the obesity market segments among patients “looking for maybe different depths of weight loss or different tolerability profiles.”Meanwhile, Roche has yet to make a final call on whether to take the GLP-1/GIP receptor agonist CT-388—another Carmot asset—into a phase 3 obesity trial, but Graham said the company is “right on the cusp” of taking that program forward.“We plan to make those decisions later this year, and, once we get rolling here, I think you\'re really going to see us flesh out our [obesity] strategy,” she said. When it comes to bringing new drugs into Roche’s pipeline, Graham was tight-lipped on which of the company’s five core focus areas—neurology, oncology/hematology, immunology, ophthalmology and CVRM (cardiovascular renal metabolic)—the company is prioritizing for M&A.“Clearly, within our five therapeutic areas, there are definitely places where we would like to bring in additional assets,” she told Fierce. “As Thomas [Schinecker] said, we look at an unbelievably large number of deals every year, and are constantly on the hunt, not only for things that will fit that strategy but things that are truly transformational.”With such a multifaceted pipeline, what are the readouts Graham is most excited about?“It\'s like trying to choose your favorite child,” she said, before name-checking the recent “super exciting” phase 2 results for the BTK inhibitor fenebrutinib in relapsing multiple sclerosis (MS). “It could be a really huge step forward for MS patients,” she explained.There’s also the “high-risk, high-reward bet” on the Parkinson’s drug prasinezumab, said Graham, who ended by referencing the IgG1 monoclonal antibody afimkibart.Along with CT-388 and the Alzheimer’s disease prospect trontinemab, afimkibart was one of the assets Roche fast-tracked as part of its reallocation of R&D resources.“I\'m an immunology girl at heart—it’s where I started my career,” Graham explained. “There\'s so much opportunity in the immunological space and so many diseases that we could potentially tackle with afimki.”

1、新闻事件：2、定价和市场预测（国内及国际类似产品市场表现类比）3、观点1、新闻消息：2025年7月23日，多家媒体报道，国产创新药公司信念医药的AAV基因治疗药物波哌达可基注射液（商品名：信玖凝，研发代号：BBM-H901）定价出炉，每瓶定价9.3万元。根据说明书，该基因疗法的具体用药量，将按照患者体重计算，例如70kg体重的处方用量是44瓶，总治疗费用高达409.2万元。目前，信念医药已经发起慈善援助项目，在该项目支持下，患者用药如果超过30瓶，则只需要支付30瓶的费用，超过30瓶的部分可以获得免费赠药。但是即使如此，患者的自费部分仍然需要279万人民币。2、关于定价和销售预测关于定价：在产品上市时，增进做过预测，预计亲民点BBM-H901定价在70-80万/年，加上医保或者赠药等，价格应该差不多合理。这样每年最终能覆盖到500人，每年能有个3-5亿收入，应该是很不错了。5年一个循环（估计应该能持续有效5年吧），要是持续到8年，那就很不好意思了，5年后说不定就没人用了（有钱的都用了，还在有效期，剩下的就是没钱的）~~~这个价格比当时预测的高了4倍。CAR-T ，复星凯特120万定价，每年160张左右处方。罗氏的艾美赛珠单抗在国内费用是130万/年，2023年国内销售额只有2000万左右，BBM-H901会有多少？10张、50张，还是100张？再看看国际上其他产品表现：其他已经上市的三款血友病基因治疗均是惨淡经营。但是标价确实全球最贵：Roctavian标价290万美元，Hemgenix和Beqvez标价均为350万美元。如此高的标价下CSL的Hemgenix，2022年上市，销售额一直是个谜，在网上看到几个数据，年销售额超过了10亿美元（无法证明真伪，就简单一说）。Roctavian也是2022年上市，2023年买了360万美元，在2024年卖了2600万美元，这个和当初的预测相差甚远。根据标价推算，意味着24年只有十个患者使用了该药；23年，那只有可怜1个人；至于，Beqvez在临床试验外，实在是没有患者使用。2025年初，辉瑞宣布停止血友病B基因疗法Beqvez的全球开发和商业化，彻底放弃了该产品~。其他有意思的血友病产品可以看看下面这篇文章信念 血友病B基因治疗药物申报上市，定价预测，代表产品简述3、观点这个定价是超出了杂评君自己的预测，生产成本应该是一个不可忽视的因素。血友病B换着在国内 登记人数估计四五千人的样子。很关键一点事，血友病患者，觉大多是原生家庭条件都不好，因为这个病会影响患者的就业和生活，对家庭拖累很大。所以，这个价格应该是更有象征性意义，不会真的依靠中国市场就能赚到多少钱~出海估计也难，在美国的几个产品表现也不尽人意~但是从另一方面，还是比美国的同类药物还是便宜很多很多在海南这个地方，或者其他地方，吸引美国患者来国内治疗呢~有没有机会？这里不太熟悉跨国医保或者保险的问题，有熟悉的可以留言讨论。关于血友病介绍，下面这篇文章有一些综述，有兴趣的可以点开看看信念血友病基因治疗药物上市，狂欢之后，市场表现会如何？信念 血友病B基因治疗药物申报上市，定价预测，代表产品简述血友病领域研发全景-行业调研anti-TFPI血友病新药国内报上市，简述TFPI差异化作用机制及血友病也可以关注下知识星球，有分享一篇综述，不过是大概一年前整理的了下面是我的咨询业务，欢迎多来勾搭~~