Bristol Oy

2024年，美国食品药品监督管理局（FDA）的新药批准数量在经历了2022年的低谷和2023年的高峰后，逐渐回归到近十年的平均水平。作为一名疫苗专家，我注意到在这一年中，生物技术公司继续在新疗法和疫苗的开发中扮演重要角色，尤其是在罕见病和一些慢性疾病领域。本文将回顾2024年FDA批准的亮点，并探讨未来可能的发展趋势。一、2024年FDA批准概况 2024年，FDA共批准了54种新疗法和疫苗，其中近三分之二来自生物技术公司，而非大型制药企业。这一趋势反映了生物技术公司在商业上的雄心壮志，尤其是在罕见病药物开发方面，这些药物占据了2024年所有新药的一半以上。此外，2024年还见证了多种慢性疾病的治疗突破，包括代谢功能障碍相关脂肪性肝炎（MASH）、精神分裂症、阿尔茨海默病、慢性阻塞性肺病（COPD）和高血压等。 二、2024年批准的亮点 （一）MASH治疗的突破 2024年3月，Madrigal Pharmaceuticals的resmetirom（商品名Rezdiffra）成为首个获批用于MASH（一种与肥胖或糖尿病共病的脂肪性肝病）的疗法。这一批准标志着在寻找有效MASH药物的漫长历程中迈出了重要一步。Resmetirom是一种口服甲状腺激素受体β激动剂，尽管其完全批准还需要确认性数据，但这一突破为MASH治疗带来了新的希望。（二）中枢神经系统（CNS）药物的复兴 2024年，FDA批准了两种新的CNS药物，分别用于治疗精神分裂症和阿尔茨海默病。Bristol Myers Squibb的Cobenfy（xanomeline-trospium）是自1989年以来首个获批的新类别精神分裂症治疗药物。而Eli Lilly的donanemab（商品名Kisunla）则成为继2023年lecanemab之后第二个针对阿尔茨海默病的疾病修饰疗法。这些批准表明，针对中枢神经系统疾病的药物研发正在重新获得关注。（三）心血管疾病的新疗法 2024年，FDA批准了四种心血管疾病药物，其中Merck & Co.的sotatercept（商品名Winrevair）为肺动脉高压（PAH）治疗带来了新的机制。Sotatercept是一种融合蛋白，通过抑制activin信号通路，减少肺部血管细胞的增殖，从而减轻血管增厚。这一新机制为PAH患者提供了更有效的治疗选择。（四）罕见病药物的持续增长 2024年，超过一半的新药被归类为孤儿药（用于治疗美国患者少于20万人的疾病）。这些孤儿药包括用于治疗WHIM综合征（一种免疫系统疾病）的mavorixafor（商品名Zolremdi）和用于治疗家族性乳糜微粒血症综合征的olezarsen（商品名Tryngolza）。这些药物的批准进一步丰富了罕见病患者的治疗选择。三、2025年展望 （一）潜在的新药申请和批准 2025年，我们有望看到更多针对重大代谢性疾病的新药申请。例如，Novo Nordisk的CagriSema（semaglutide和cagrilintide的组合）以及Eli Lilly的retatrutide（GLP1/GIP/胰高血糖素受体激动剂）均处于肥胖及相关疾病的3期临床试验中。此外，Vertex的suzetrigine（一种口服NaV1.8疼痛信号抑制剂）和Sanofi的tolebrutinib（一种口服、脑穿透性BTK抑制剂）预计也将在2025年提交申请。（二）生物技术公司的崛起 2024年，生物技术公司主导了新药批准，这反映了它们在特定疾病领域的专业能力和灵活性。例如，Madrigal Pharmaceuticals的Rezdiffra和Ionis Pharmaceuticals的Tryngolza均是生物技术公司在罕见病领域的首次商业成功。这种趋势表明，生物技术公司正在逐渐成为新药开发的重要力量。（三）生物类似药的增长 2024年，FDA批准了18种生物类似药，其中包括4种可互换的生物类似药。这一增长表明，生物类似药市场正在加速发展，为患者提供了更便宜的替代选择。然而，复杂的定价动态和市场准入规则可能会限制这些药物的快速采用。四、结论 2024年，FDA的新药批准回归到较为稳定的水平，生物技术公司在新药开发中继续发挥重要作用。罕见病药物、慢性疾病的新疗法以及生物类似药的增长，为患者带来了更多的选择。随着2025年的到来，我们期待看到更多针对重大疾病的创新疗法，以及生物技术公司在商业领域的进一步崛起。作为一名疫苗专家，我特别关注生物技术公司在疫苗开发中的潜力，尤其是在应对新兴传染病和改善全球健康方面。未来，随着技术的进步和政策的支持，生物技术有望为全球健康带来更多突破。 识别微信二维码，可添加药时空小编 请注明：姓名+研究方向！

The U.S. Food and Drug Administration (FDA) announced on Friday the approval of Bristol Myers Squibb's (BMS) injectable cancer drug Opdivo Qvantig. Opdivo, a PD-1 inhibitor, works by blocking the immune system’s natural inhibitory mechanisms, enhancing its ability to attack cancer. Previously, Opdivo was only available through intravenous infusion, requiring patients to undergo IV administration at medical facilities. The new injectable form is expected to provide convenience for patients and help protect the company’s sales as patents for the IV version near expiration. Branded as Opdivo Qvantig, the new formulation has been approved for all previously authorized adult solid tumor indications and can be used alone or in combination with chemotherapy. Before FDA approval, Bristol’s Chief Commercialization Officer Adam Lenkowsky told Reuters that the drug is expected to launch in early January, with pricing equivalent to the IV version. The IV version is priced at $7,635 per low-dose injection for two weeks and $15,269 for 480 mg per injection every four weeks. The approval was based on late-stage study data showing that the subcutaneous version of Opdivo is comparable in effectiveness to the IV form in patients with advanced kidney cancer who had received prior systemic therapy. Given the upcoming patent expirations of older drugs like cancer treatment Revlimid and blood thinner Eliquis, Bristol Myers Squibb is relying on new therapies like Opdivo Qvantig to drive growth. Co-developed with Halozyme Therapeutics, Opdivo Qvantig employs a novel drug delivery technology that reduces treatment time from hours of infusion to just minutes of subcutaneous injection.

Bristol Myers Squibb is ending a pair of cell therapy collaborations with Immatics and nixing a separate agreement with Century Therapeutics for natural killer cell and T cell-based cancer treatments. These appear to be the latest changes resulting from Bristol Myers’ detailed plans to save $1.5 billion , including from pipeline cuts, announced in April. BMS and Immatics started working together in 2019, when the pharma company spent $75 million upfront on options for three of the biotech’s T cell receptor therapy targets. Two years later, BMS licensed a TCR bispecific called IMA401 for $150 million upfront, only to hand back the rights this September, with Immatics losing out on $770 million in potential milestones for the Phase 1 candidate. In the most recent blow, Bristol Myers ended two expansions to their original 2019 pact, according to an SEC filing published Friday. The first extension , dating back to 2022, was focused on developing gamma delta allogeneic cell therapies and featured $60 million upfront and up to $700 million in milestones per program. The second deal saw BMS exercise an option for an unnamed TCR therapy in 2023, and included $15 million and up to $490 million in milestones. The duo’s 2019 agreement technically remains in place, according to the filing. As for Century, the Philadelphia-based drugmaker secured a licensing deal with BMS that featured $150 million in a combination of cash payments and equity investment in 2022. The agreement centered on up to four off-the-shelf cell therapies made using donor stem cells. But Bristol has now nixed the deal entirely, according to an SEC filing published Thursday, with Century losing out on up to $3 billion in milestones. Century said in the filing it is still “encouraged” by the programs that were partnered with BMS, and will look at potential opportunities for advancement in acute myeloid leukemia and multiple myeloma.