A 4-Week, Phase II, Multicenter, Randomized, Double-Masked, Vehicle-Controlled, Parallel Group Study With 4 Weeks of Follow-Up to Evaluate Safety and Efficacy of a new formulation of Recombinant Human Nerve Growth Factor (rhNGF) Eye Drop Solution at two different Concentrations in patients with Dry Eye Disease (REDUCO study) - NGF0123

开始日期2024-05-17

申办/合作机构-

NCT06411145

/ Withdrawn临床4期

A Prospective, Multicenter Trial to Evaluate Efficacy and Safety of Cenegermin-bkbj (20 mcg/mL) Ophthalmic Solution on Corneal Thickness Via Anterior Segment Optical Coherence Tomography (AS-OCT) in Stage 3 Neurotrophic Keratitis

Primary Objective:
To assess the efficacy of cenegermin-bkbj (20 mcg/mL) ophthalmic solution on overall corneal thickness via AS-OCT in patients with stage 3 neurotrophic keratitis with respect to change from baseline at weeks 4, 8, and 16.
Secondary Objectives:
To assess the effects of cenegermin-bkbj (20 mcg/mL) ophthalmic solution on corneal stromal thickness via AS-OCT in patients with stage 3 neurotrophic keratitis with respect to change from baseline at weeks 4, 8, and 16.
To assess the effects of cenegermin-bkbj (20 mcg/mL) ophthalmic solution on corneal stromal reflectivity via AS-OCT in patients with stage 3 neurotrophic keratitis with respect to change from baseline at weeks 4, 8, and 16.
To assess the effects of cenegermin-bkbj (20 mcg/mL) ophthalmic solution on corneal sensitivity via Cochet-Bonnet aesthesiometer in the center of the lesion with respect to change from baseline at weeks 4, 8, and 16.

开始日期2024-04-26

申办/合作机构

Dompe Farmaceutici SpA

NCT06244316

/ Completed临床2期

A 4-Week, Phase II, Multicenter, Randomized, Double-Masked, Vehicle-Controlled, Parallel Group Study With 4 Weeks of Follow-Up to Evaluate Safety and Efficacy of a New Formulation of Recombinant Human Nerve Growth Factor (rhNGF) Eye Drop Solution at Two Different Concentrations in Patients With Dry Eye Disease

The purpose of this study is to evaluate the safety and efficacy of two different concentrations of the new formulation of rhNGF ophthalmic solution versus vehicle, in order to demonstrate superiority of at least one of the two concentrations over vehicle in the improvement of ocular symptoms of dry eye in participants with dry eye disease (DED). The rhNGF ophthalmic solution, or vehicle, will be administered as one drop in each eye, three times a day, for 4 weeks. Participants will attend a total of 5 study visits from screening through end of the study (Week 8), which will include eye exams and questionnaires.

开始日期2024-01-22

申办/合作机构

Dompe Farmaceutici SpA

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项与塞奈吉明相关的文献（医药）

2025-12-01·EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES

Safety and pharmacokinetics of escalating doses of recombinant human nerve growth factor eye drops (HT006.2.2) in healthy subjects

Article

作者: Hu, Xiaojun ; Ni, Siyang ; Lu, Laichun ; Jie, Ying ; Lu, Huan ; Pang, Hongyan ; Tang, Huadong ; Wu, Feng ; Yu, Wei ; Jiao, Ying ; Dai, Yuyang ; Han, Ying

BACKGROUND:

HT006.2.2 is a kind of recombinant human nerve growth factor (rhNGF) eye drop with the potential to treat neurotrophic keratitis.

OBJECTIVE:

To evaluate the safety, immunogenicity, and pharmacokinetic (PK) characteristics of HT006.2.2 eye drops in Chinese healthy volunteers.

METHODS:

This study was divided into three parts. In Part A (single-dose) and Part B (multi-dose), four dose groups were per-specified: 5 µg/mL, 20 µg/mL, 60 µg/mL and 120 µg/mL. Each dose group comprised eight subjects randomized in a 3:1 ratio to receive the HT006.2.2 or placebo. Serum samples were collected for systemic PK analysis and anti-drug antibody (ADA) detection. The Part C comprised two single-dose groups (20 µg/mL and 60 µg/mL), and tear samples were collected at preset time points for tear fluid PK analysis. The drug mass per gram of tears (unit: µg/g) was calculated and used as the drug concentration to recalculate the PK parameters. Safety was evaluated via vital signs monitoring, 12-lead electrocardiogram, clinical laboratory tests, and ophthalmologic assessments (ocular symptoms and signs, slit-lamp microscopy, corneal fluorescein sodium staining, intraocular pressure measurement, and best-corrected visual acuity). Adverse events were collected following the initial administration of the investigational drug and were defined as treatment-emergent adverse events (TEAEs).

RESULTS:

This study enrolled a total of 163 subjects, with 161 receiving the medication and completing the study as planned. HT006.2.2 showed favorable tolerance across both single-dose and multiple-dose parts. At the highest dose of 120 µg/mL, no maximum tolerated dose (MTD) was reached. No discernible dose-response relationship was identified in the incidence of TEAEs across dosage groups, with the total TEAE rate in HT006.2.2 groups remaining non-exceeding that of the placebo group. Ocular TEAEs included corneal vital dye staining, eye itching, eye pain, conjunctival hyperemia, and decreased visual acuity. In addition, no HT006.2.2 or ADA was detected in the peripheral blood of healthy subjects. Tear fluid PK analysis of HT006.2.2 (20 µg/mL) showed time to peak concentration (T_max) of 0.17 h, mean peak concentration (C_max) of 3.63 ± 2.06 µg/g, elimination half-life (t_1/2) of 4.21 h.

CONCLUSIONS:

HT006.2.2 eye drops exhibited favorable safety in Chinese healthy subjects, with localized ocular distribution, no systemic exposure, and no detectable ADAs. These findings support the continuation of clinical development for HT006.2.2 in China.

2025-10-01·Ocular Surface

Prospective consecutive case series of patients with neurotrophic keratopathy associated with unilateral total limbal stem cell deficiency caused by severe ocular surface burns

Article

作者: Figueiredo, Francisco C ; Mudhar, Hardeep-Singh ; Lako, Majlinda ; Stefanache, Teodor ; Baylis, Oliver J ; Figueiredo, Gustavo S

INTRODUCTION:

Limbal stem cell deficiency (LSCD) is commonly caused by ocular surface burns. Corneal nerve damage, often due to infection, inflammation, trauma, leads to neurotrophic keratopathy (NK). This study investigates corneal sensation and nerve damage in patients with unilateral total LSCD before and after autologous cultivated limbal epithelial transplantation (Auto-CLET).

METHODS:

This prospective, single-centre case series included patients with unilateral total LSCD due to severe burns treated at Royal Victoria Infirmary, UK, between June 2012 and January 2018. Corneal sensation was assessed using Cochet-Bonnet aesthesiometry at baseline and 6 months post-Auto-CLET, and in vivo confocal microscopy (IVCM) was used to examine nerve regeneration.

RESULTS:

Twenty-three patients (19 males, 4 females) received Auto-CLET. Successful restoration of the ocular surface was achieved in all patients, confirmed by slit lamp examination, impression cytology, and IVCM at follow-ups up to 36 months. Baseline best corrected visual acuity was 1.67 logMAR in the affected eye compared to a mean of -0.08 in the healthy eye. Corneal aesthesiometry in LSCD eyes averaged 9.13 mm at baseline, increasing to 12.0 mm at 6 months post-Auto-CLET, though this change was not statistically significant (p = 0.26). IVCM revealed no regeneration of corneal nerves at any follow-up intervals, indicating persistent nerve damage.

CONCLUSIONS:

Despite successful ocular surface restoration, corneal nerve damage persisted, leaving patients at risk of persistent NK. To address this, we strongly suggest continuous topical treatment with blood derived products, such as serum eye drops, but also newly developed therapies such as IGF and rhNGF class of drugs.

2025-08-06·ACS Chemical Neuroscience

Selective Modulation of Trk Receptors by Cyclo-Organopeptides

Article

作者: Mi, Tianxiong ; Pewklang, Thitima ; Lyu, Rui-Liang ; Thompson, Tye ; Sefiani, Arthur ; Burgess, Kevin ; Kamkaew, Anyanee ; Joy, Shaon

Neurotrophins (NTs, including NGF, BDNF, NT-4, and NT-3) are extracellular cytokines which modulate the survival and growth of cells expressing tropomyosin receptor kinases (Trks) A-C. Cells which express Trks include many neural tissues. For instance, corneal nerves secrete NTs to counteract epithelium disruption. Potential therapeutic applications of Trk agonism are numerous, but the use of NTs is limited by problems with production, in vivo stability, and side effects of the protein. Only humanized recombinant NGF has been clinically approved: Cenegermin for treatment of neurotrophic keratitis (NK) in the eye. Consequently, low molecular mass Trk agonists are of interest as surrogates for humanized NTs. One low molecular mass TrkA modulator from our lab, a cyclo-organopeptide D3, emerged as a clinical candidate for treatment of dry eye disease and reached phase 3 trials. However, it remains to be determined whether similar agonists or modulators of other Trks might exhibit similar effects. Moreover, D3 was moved into trials without much optimization. This work was undertaken to identify cyclo-organopeptides which would activate TrkA, B, and/or C and to compare their potencies to D3. The immediate goal was to select compounds for studies to probe relief from desiccating stress to the eye in a mouse model relative to D3. In fact, in vivo assays on select compounds developed in the work described here have already been published. Three new cyclo-organopeptides selected for Trk agonism or modulation and D3 were tested, and a superior lead for relief of desiccating stress in vivo was identified. Interestingly, that lead compound was designed to mimic NT-3, not NGF. This paper describes how those new cyclo-organopeptides were designed, prepared, and then selected via screens on Trk-transfected cells. It also outlines and explains obstacles which limit progress in this type of study.

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·BiG生物创新社

FDA发新券，审评缩至1个月！

作者｜YY 2025年10月16日，美国食品药品监督管理局（FDA）宣布了“局长国家优先券”（CNPV, Commissioner’s National Priority Vouchers，简称“国家优先券”）试点项目首批九家药企及其在研项目。这是FDA在原有优先审评券（Priority Review Voucher, PRV）制度之外新增的一种“国家优先”版本的制度，被视为对审评资源重新分配的一次制度试验。 FDA在今年6月正式推出这一试点项目，其目的在于奖励那些“符合国家卫生优先事项”的企业或产品。与传统的罕见儿科疾病、热带病和生物威胁应对的三类PRV覆盖范畴不同，国家优先券并不限定具体疾病领域，而是将适应症的公共卫生意义、生产布局及社会价值纳入考虑范围。根据FDA公布的说明，持券药物的审评周期可从常规的10-12个月压缩到约1至2个月，并享受更紧密的监管沟通。 01 认证一张，黄金万两在FDA的审评体系中，药品能以多快的速度获批，往往决定了它能否在市场上领先一步，享受更长的市场独占期。常规的新药审评周期约为十至十二个月，这一标准审评流程主要针对大多数非急迫性药物。若药物被授予优先审评（Priority Review）资格，将使审评时间从常规的十至十二个月缩短至六个月。 2007年，美国国会进一步建立了“优先审评券（PRV）”制度。它并不直接授予某个药物加速资格，而是授予开发者一张“可用于未来任意药品”的加速券，即PRV。企业在获得这张券后，可以为任何新药申请优先审评，将审评期同样缩短至六个月。PRV最初用于激励企业开发被忽视的热带病、罕见儿科疾病及生物威胁防治药物。而2025年推出的“国家优先券（CNPV）”，在速度与范围上都迈出了更大一步。它承诺将审评周期压缩至仅一至两个月，并不限于特定疾病类型。只要药物被认为符合“国家健康优先事项”，或者能增强美国药品制造能力、提高可负担性，就有资格申请。换言之，CNPV不再只是科研激励，更是一项兼具公共卫生与产业战略导向的制度创新。在制药业，一天的时间差往往意味着上百万美元的收入差距。优先审评券（PRV）之所以被称为“黄金券”，并非比喻，而是可以被精确计算的现实数字。以年销售额10亿美元的“重磅炸弹”药物为例，折算到每日收入约为274万美元。如果企业能通过CNPV将上市时间从常规的10个月缩短至仅1至2个月，就意味着可以提前约240天（约8个月）进入市场。这240天的优势如果发生在重磅炸弹类型的药物身上，对应的潜在销售额约至少将为6.58亿美元；若药物的年销售额为20亿美元，则意味着超过13亿美元的额外收益。这还只是账面上的直接回报，未计入市场先发优势、竞争排他期延长及资本估值提升等连锁效应。这张“时间券”的回报率在现实案例中已被验证。礼来的2型糖尿病药物Mounjaro于2022年凭借一张优先审评券提前四个月上市。公司并未披露所用凭证的具体来源，但普遍认为是其在2018年以约8000万美元购得的一张PRV。8000万美元换来十几亿美元的季度销售回报，使PRV成为名副其实的“黄金万两”。更关键的是，Mounjaro既不属于热带病，也非罕见儿科疾病，更与生物威胁防治无关，却享受到了与公共卫生急需药物相同的加速待遇。从企业角度看，这张券的吸引力无可比拟：在资本驱动的研发体系中，能让药物提前几个月上市的制度，有可能会为企业及其产品享受更多的真正市场独占期，从而带来巨大的利润。根据现有统计，至少有八款获得PRV的药物后来成为年销售额超过10亿美元的重磅产品，包括辉瑞的Paxlovid和Comirnaty、Moderna的Spikevax、Vertex的Trikafta、吉利德的Veklury、Biogen的Spinraza、诺华的Zolgensma以及罗氏的Evrysdi。它们的存在证明，优先审评券已不再局限于激励冷门药物的工具，而成为进入重磅市场的加速器。这些数据解释了为何当FDA宣布推出CNPV，一项承诺将审评周期压缩至仅1至2个月的新计划时，整个行业都为之震动，这意味着政策杠杆的力度再次升级。 02 九家首获CNPV黄金券在首批九张“国家优先券”的名单中，既包括在传统创新领域具有突破性的药物，也包括被视为契合国家战略目标的生产型项目，显示出FDA在科学创新与产业安全之间寻求平衡的意图。德国默克旗下的EMD Serono成为外界关注的焦点。其生育药物Pergoveris是一种重组促卵泡激素与促黄体生成素的复方制剂，能够刺激卵泡发育，用于辅助生殖治疗。该药已在全球74个国家上市，但在美国尚未获得批准。白宫宣布与 EMD Serono 达成大幅降价与直售安排；公司同时表示将按 CNPV 路径申报 Pergoveris。两项举措并行，体现 CNPV 将“药品可负担性”与国家利益纳入考量。此举体现了CNPV制度将“药品可负担性”纳入遴选考量的导向。另一家入选的跨国药企是赛诺菲。其单克隆抗体Teplizumab用于延缓1型糖尿病的发病进程，目标人群为处于疾病早期阶段、尚能分泌部分胰岛素的患者。1型糖尿病是典型的自身免疫疾病，目前尚无治愈手段。该药此前已被FDA批准用于高风险人群的预防性治疗。此次获券的理由，是其对未被满足的代谢疾病需求具有潜在的公共卫生意义。FDA在说明中指出，1型糖尿病是“极具社会负担但治疗进展有限的领域”，因此被纳入国家卫生优先事项。 Regeneron的基因疗法DB-OTO是名单中技术含量最高的项目之一。该疗法针对一种罕见的遗传性耳聋，通过基因递送修复听觉功能。《新英格兰医学杂志》披露的结果显示，12 例中 11 例出现临床意义的改善，其中3 例达到正常听力；试验按预设主要终点要求9 例改善且已达成。这类罕见病基因疗法代表了FDA支持前沿技术加速转化的取向。监管方表示，选择DB-OTO的理由在于“其为首批可恢复感官功能的基因疗法之一，对残疾群体具有重大影响”。意大利的Dompé Farmaceutici凭借Cenegermin-bkbj获得一席。这是一种重组人神经营养因子，用于治疗神经性角膜炎（neurotrophic keratitis），该病可导致角膜损伤甚至失明。Cenegermin-bkbj此前已在美国获批用于这一罕见适应症，但此次列入名单的原因，是FDA认为该药“可代表视觉功能康复领域的重要治疗方向”，反映出监管部门对恢复性疗法的重视。来自美国的Revolution Medicines提交的RMC-6236是一种靶向胰腺癌的候选药物。该公司在去年公布的1期研究中报告了积极结果，显示对部分KRAS突变患者的临床获益。FDA将其纳入CNPV名单，是出于“在高致死率肿瘤中加快潜在创新治疗的考量”。胰腺癌长期被认为是抗癌药开发中最具挑战性的领域之一。 Disc Medicine的Bitopertin则代表了血液罕见病方向。该药用于治疗卟啉病（porphyria），这是一种导致光敏性极强、影响皮肤及肝脏功能的罕见代谢病。公司在9月提交了加速批准申请。FDA指出，该药物能够“改善长期被忽视的罕见代谢性疾病患者的生活质量”，因而获得优先审评券。 Achieve Life Sciences的Cytisinicline是一种源自植物的戒烟辅助药，靶向尼古丁成瘾的神经通路。与传统尼古丁替代疗法不同，它以非尼古丁方式缓解戒断反应。FDA将其列入名单的理由之一，是“应对电子烟使用人群迅速上升的公共健康挑战”。在名单的最后，两家美国本土制造企业的入选，即Phlow Corporation与US Antibiotics，反映了制度设计的另一层意图。Phlow是一家在疫情期间承接政府合同、致力于恢复美国仿制药制造能力的公司，此次凭借麻醉药物氯胺酮（Ketamine）获得CNPV；US Antibiotics则因生产复方抗生素Augmentin XR（阿莫西林和克拉维酸钾）而获券。FDA在新闻稿中提到，这两项授予旨在支持国内制药供应链的稳定性，显示出CNPV制度将“制造业回流”视为与创新同等重要的评估维度。 FDA局长Marty Makary在社交媒体视频中表示，获券项目的提名由各药品审评中心提出，最终由多学科团队评审决定。评审依据包括疾病负担、患者可及性、以及产品对美国卫生体系的战略价值。他强调，这些产品将获得“更紧密的沟通和更快的决定”，但“加速并不等于放宽标准”。 03 从PRV到CNPV 制度的延展与再定义如果说这些入选项目勾勒出“国家优先券”所覆盖的边界，那么它们背后的政策逻辑，正是FDA在传统PRV框架上作出的主动延展。 “优先审评券（PRV）”制度起源于2007年，当时美国国会通过法案，授权FDA对开发被忽视的热带病药物的企业给予可转让的审评加速凭证。随后，这一机制被扩展至罕见儿科疾病和生物威胁防治药物领域。它的核心机制是：当一家企业在这些领域获得FDA批准后，可获得一张PRV，用于任何后续药物申请，使审评周期由约10个月缩短至6个月。企业既可自用，也可转售，形成了一个独特的政策市场。随着时间推移，这些凭证的交易价格一度达到数亿美元。2015年，艾伯维以3.5亿美元的价格从United Therapeutics购得一张PRV，加速类风湿性关节炎药物Rinvoq的适应症审查。这类交易表明，PRV已成为“时间即金钱”的象征，也逐渐偏离了当初为鼓励冷门领域研发而设立的初衷。在此背景下，CNPV的推出显然带有重新校准政策重心的意图。首先，CNPV是不允许转让的；其次，它不再仅限于治疗被忽视疾病的激励，而是试图让FDA的审评加速资源，与更广义的“国家卫生与产业优先级”对齐。从Pergoveris的价格承诺，到Phlow与US Antibiotics的本土生产，再到Teplizumab和DB-OTO代表的临床创新，CNPV的首批名单实际上构成了一张政策地图，显示出监管机构如何在激励创新、保障可负担性与维护供应链安全之间寻找新的平衡点。此次“国家优先券”是PRV体系的进一步衍生，与以往不同之处在于，除疗效与疾病负担外，FDA首次将产业政策因素纳入考量。部分获券项目与美国药品本地化生产、供应链安全直接挂钩。例如Phlow和US Antibiotics的获选理由，正是其在美国境内生产仿制药，符合“制造业回流”的战略要求。但这一新的遴选维度也引起部分监管观察者的担忧。报道指出，FDA在近两年经历了超过3500人的裁员与离职，外界担心加速审评可能加重审评团队负担，影响审评质量。另有评论指出，该计划的遴选标准相对模糊，容易被政治意图或商业游说所左右。 FDA方面在声明中回应称，获券药物仍需通过完整的安全性与有效性审查流程，审评团队将采用“肿瘤委员会式”的跨学科会议机制集中评议，确保快速但不草率。对于不完整申请或存在生产问题的项目，FDA仍可延后审评进度。结语从热带病和罕见儿科疾病，到国家卫生优先事项，优先审评券制度的演变，映射了FDA在不同历史阶段的政策取向。它曾是为了让资本流向被忽视的医学空白，如今却成为影响整个产业节奏的制度杠杆。PRV和CNPV之间的差别，不只是审评速度的不同，更是价值分配逻辑的重构：谁能更快获批，谁就能先进入市场，谁就能占据时间的红利。这种加速无疑让患者更早获得治疗，也让企业收获了更高的商业回报。时间与价值被放在同一坐标轴上，政策激励与市场动力开始互为放大。然而，随着审评通道的层级化加深，如何确保资源分配仍以临床需求为中心，而非被商业利益牵引，正在成为新的考验。监管者希望通过CNPV激励创新、保障供应链、强化产业韧性，但每一次“提速”也都意味着更高的审评压力、更大的透明度要求。制度的成功，不仅取决于它能否让药物更快抵达患者，更取决于它是否能让社会信任这种“速度”的正当性。 CNPV也许是一个新的起点。它让人重新思考：在一个以科学、资本与政策共同驱动的行业里，什么样的速度，才是真正值得被奖励的。 Ref. FDA Awards First-Ever National Priority Vouchers to Nine Sponsors. FDA. https://www.fda.gov/news-events/press-announcements/fda-awards-first-ever-national-priority-vouchers-nine-sponsors Sibbesen, J. B. et al. Understanding the FDA Approval Process and PDUFA Dates. Pharmacy Times. 20. 03. 2025. Priority Review. FDA. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/priority-review CENTER FOR DRUG EVALUATION AND RESEARCH. APPLICATION NUMBER: 215866Orig1s000. SUMMARY REVIEW DB-OTO Results in the New England Journal of Medicine Showcase Dramatic and Sustained Improvements in Hearing and Speech Perception in Children with Profound Genetic Hearing Loss. Regeneron Press Release. 12. 10. 2025. 共建Biomedical创新生态圈！如何加入BiG会员？

优先审批加速审批快速通道申请上市突破性疗法

2025-10-20

·Pharmaceutical Technology

Infertility treatment heads first drugs on FDA’s new priority review scheme

Pharma companies wishing to reap the benefits of the programme need to demonstrate US-centric potential for their drug candidates. kemalbas via GettyImages. The US Food and Drug Administration (FDA) has selected the first products to be included on the Commissioner’s National Priority Voucher (CNPV) pilot programme. A total of nine drug candidates have joined the scheme launched in June 2025 , which will see its developers receive a voucher that will significantly expedite their path to approval. The group is headed by Merck’s Pergoveris (follitropin alfa/lutropin alfa), which is already approved in Europe, for the treatment of infertility. President Donald Trump specifically identified Pergoveris’ inclusion, touting the drug’s potential to offer a cheaper option to US patients. “This drug would directly compete against a much more expensive option that currently has a monopoly in the American market, and this will bring down costs very significantly,” Trump said during an IVF news conference at the White House. Trump was likely referring to Gonal-f, made by Merck’s US and Canadian healthcare business EMD Serono. Gonal-f generated global sales of €833m in 2024. The pharma company plans to use its CNPV to file for FDA approval of its new drug, which it disclosed while announcing a broader deal with the US government to expand fertility access treatment. Elsewhere on the list, candidates being developed for hearing and vision loss have been included in the form of Regeneron’s gene therapy DB-OTO and Dompé Farmaceutici’s Oxervate (cenegermin-bkbj), respectively. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence Sanofi’s teplizumab, which is already FDA-approved to delay the onset of stage 3 type 1 diabetes, has been included on the list. The agency said it was selected for “type 1 diabetes,” hinting that the review could be for a broader label approval. Revolution Therapeutics’ RMC-6236, in development for the treatment of pancreatic cancer, and Disc Medicine’s porphyria therapy bitopertin round off the list of products for specific diseases. The FDA selected cytisinicline for nicotine vaping addiction, while ketamine and Augmentin XR were chosen to boost domestic manufacturing of anaesthesia and antibiotics, respectively. The CNPV programme is the latest move by the Trump administration to bolster its pharmaceutical armoury and incentivise the development of new drugs specifically for Americans . Pharma companies wishing to reap the benefits of the programme need to demonstrate US-centric potential for their drug candidates. Therapies need to address a health crisis in the country, produce an innovative cure for American patients, meet public health needs, or increase domestic drug manufacturing as a national security issue. In its statement sharing the first batch of recipients, the FDA said that each product has significant potential to address one or more of these major national priorities. The agency stated that it would likely add another group of CNPV recipients in the coming months. Companies with a product in the programme will receive a decision within 1-2 months, compared to the usual 10-12 months, following filing of a complete application for a drug or biologic. Sponsors will also be able to undertake enhanced feedback with FDA staff during development and the review window. “One of our core goals is to deliver more cures and meaningful treatments – especially ones that have an outsized impact on our most pressing national priorities. We must modernise the review process and try new approaches to meet the needs of the American people,” said FDA commissioner Marty Makary. While the CNPV pilot begins its journey in the US regulatory landscape, experts have been concerned that its activity could take attention away from similar programmes that incentivise rare diseases. Pharmaceutical Technology Excellence Awards - The Benefits of Entering Gain the recognition you deserve! The Pharmaceutical Technology Excellence Awards celebrate innovation, leadership, and impact. By entering, you showcase your achievements, elevate your industry profile, and position yourself among top leaders driving pharmaceutical advancements. Don’t miss your chance to stand out—submit your entry today! Nominate Now

上市批准优先审批

2025-10-17

·FierceBiotech

FDA names 9 first recipients of 1- to 2-month priority review vouchers

Revolution Medicines and Disc Medicine are among the recipients of a Commissioner’s National Priority Voucher. \n The FDA has handed out the first nine national priority vouchers, positioning companies including Merck KGaA, Regeneron and Sanofi to complete the approval process in one to two months.FDA Commissioner Marty Makary, M.D., launched the program in June, and the agency began accepting applications in July. The program is designed to cut product approval times, which are typically 10 to 12 months, for medicines that align with U.S. national priorities. Specific priorities include addressing the U.S. public health crisis, treating large unmet medical needs and delivering transformative treatments.The first nine recipients of a Commissioner’s National Priority Voucher (CNPV) are a mix of approved drugs and investigational medicines. The investigational products include DB-OTO, the gene therapy that Regeneron is developing to treat a rare genetic form of hearing loss. Regeneron plans to seek FDA approval this year. If the CNPV works as intended, the timeline suggests the gene therapy could come to market early next year at the latest. The company has published data on the gene therapy in 12 patients, nine of whom only received the treatment in one ear. Talking on an FDA podcast, Mallika Mundkur, M.D., who leads the CNPV program at the agency, said the DB-OTO data are “pretty remarkable” but cautioned that “we need to learn more about” its safety. The podcast was hosted by Makary, who said the hearing restoration data point to the need for the FDA “to get a decision out promptly using the standard scientific process, cutting any wasted time.” Makary added that “we\'re hearing the company may make it available either at an extremely low price or for free, which of course is part of a national priority to make drugs more affordable.” The criteria for the CNPV program include lowering the U.S. price of a drug consistent with most-favored nation pricing.Revolution Medicines’ RMC-6236 and Disc Medicine’s bitopertin are among the other recipients of a CNPV. RMC-6236, also called daraxonrasib, is a RAS-MULTI(ON) inhibitor that is in phase 3 development as a treatment for forms of pancreatic and non-small cell lung cancer. Data from one of the trials are due next year. Revolution is holding off on adjusting its timelines while it assesses the impact of the voucher.Discussing RMC-6236 on the podcast, Makary said the FDA’s oncology division “thought this is something that is potentially promising.” The FDA commissioner went on to say the agency is going into its divisions and asking reviewers what programs they think “may potentially look amazing.” Makary framed the talks as a push for the FDA to move beyond “receive-only mode” when identifying assets it can accelerate. Meanwhile, Disc\'s bitopertin is a GlyT1 inhibitor being assessed in rare, genetically driven hematologic diseases. The biotech filed for accelerated FDA approval of the small molecule last month. Disc originally applied for a priority review that would reduce the review time from 10 months to six months. The FDA also awarded CNPVs to Merck’s pergoveris and Sanofi’s teplizumab. Pergoveris, a fertility drug, is already available in 74 countries. Merck plans to file for FDA approval of the drug using its CNPV, which it disclosed as part of a broader deal with the U.S. government to expand access to fertility treatment. Teplizumab, which Sanofi sells as Tzield, is FDA-approved to delay the onset of forms of Type 1 diabetes.Officials gave another two CNPVs to cytisinicline for nicotine vaping addiction and cenegermin-bkbj for blindness. Achieve Life Sciences is seeking approval of cytisinicline for smoking cessation. Cenegermin is already sold as Oxervate by Dompé Farmaceutici to treat a corneal disease. The final two CNPVs are focused on onshoring, with the FDA using the vouchers to try to boost domestic production of ketamine for general anesthesia and of the antibiotic augmentin XR. Phlow received the ketamine CNPV.

临床3期优先审批上市批准

100 项与塞奈吉明相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	塞奈吉明	S01XA24	DB13926

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适应症	国家/地区	公司	日期
神经营养性角膜炎	欧盟	Dompe Farmaceutici SpA	2017-07-06
神经营养性角膜炎	冰岛	Dompe Farmaceutici SpA	2017-07-06
神经营养性角膜炎	列支敦士登	Dompe Farmaceutici SpA	2017-07-06
神经营养性角膜炎	挪威	Dompe Farmaceutici SpA	2017-07-06

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适应症	最高研发状态	国家/地区	公司	日期
干眼综合征	临床3期	美国	Dompe Farmaceutici SpA	2022-01-19
干眼综合征	临床3期	意大利	Dompe Farmaceutici SpA	2022-01-19
干燥综合征	临床3期	美国	Dompe Farmaceutici SpA	2022-01-19
干燥综合征	临床3期	意大利	Dompe Farmaceutici SpA	2022-01-19
干燥综合征性角膜结膜炎	临床2期	美国	Dompe Farmaceutici SpA	2017-01-20
白内障	临床2期	意大利	Dompe Farmaceutici SpA	2017-01-12
失明	临床2期	意大利	Dompe Farmaceutici SpA	2015-05-01
黄斑水肿	临床2期	意大利	Dompe Farmaceutici SpA	2015-05-01
视网膜色素变性	临床2期	意大利	Dompe Farmaceutici SpA	2015-05-01
角膜溃疡	临床2期	法国	Dompe Farmaceutici SpA	2013-01-01

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03982368 (Pubmed) 人工标引	临床2期	干眼综合征	-	Cenegermin 20 mcg/mL t.i.d.	糧積蓋繭鏇遞選鏇簾簾(鹽範鏇遞簾積壓鬱醖鏇) = 獵廠繭鹹餘糧繭鑰顧築衊齋廠廠糧製築蓋網膚 (糧選構積製膚艱糧壓窪 ) 更多	不佳	2024-07-17
				Cenegermin 20 mcg/mL b.i.d.	糧積蓋繭鏇遞選鏇簾簾(鹽範鏇遞簾積壓鬱醖鏇) = 繭製獵範願獵鑰構衊積衊齋廠廠糧製築蓋網膚 (糧選構積製膚艱糧壓窪 ) 更多
NCT04485546 (DEFENDO, Pubmed)	临床4期	神经营养性角膜炎	37	Cenegermin 20 mcg/ml	壓繭築膚憲選鏇窪壓廠(鏇簾繭艱夢夢醖積蓋窪) = 鹽淵蓋廠艱鏇鬱簾鹽積顧夢遞選鬱選餘膚簾廠 (構糧廠膚淵遞鹽繭網願, 5.1 ~ 31.9) 更多	-	2024-02-01
NCT04485546 (DEFENDO, CTgov)	临床4期	神经营养性角膜炎	37	cenegermin-bkbj	鏇製網蓋築襯憲鹹繭淵 = 衊壓顧遞餘壓鹽壓遞製簾遞蓋膚夢鹽觸糧憲淵 (壓糧網鑰鹽淵鏇膚願蓋, 淵鑰觸願蓋遞積餘艱衊 ~ 構廠壓壓膚鑰鏇繭築憲) 更多	-	2023-06-23
NCT02855450 (NGF-Glaucoma, CTgov)	临床1期	青光眼	60	rhNGF (rhNGF)	選齋壓獵齋衊淵鹹鬱願 = 鏇鹽鹹蓋鬱襯窪鏇積鬱壓鏇艱繭遞選鏇襯糧觸 (鬱夢餘鏇鹽窪廠艱膚鬱, 夢壓壓網簾夢襯選夢繭 ~ 範顧築鹽夢顧簾積觸製) 更多	-	2020-11-25
				Vehicle (Vehicle)	選齋壓獵齋衊淵鹹鬱願 = 築製廠襯衊遞製鑰鹽鏇壓鏇艱繭遞選鏇襯糧觸 (鬱夢餘鏇鹽窪廠艱膚鬱, 夢選糧遞襯積艱鑰築構 ~ 鏇製蓋鏇築簾積淵顧糧) 更多
NCT03836859 (CTgov)	临床1期	-	30	rhNGF 20μg/mL (rhNGF 20μg/mL)	遞襯構觸製衊壓構淵築 = 鏇憲淵觸製製淵鹹鑰遞繭製簾鏇衊顧衊膚膚遞 (憲製積願淵膚網壓餘壓, 夢膚顧築衊顧築鹽廠願 ~ 鏇積顧夢範壓廠膚構範) 更多	-	2020-06-11
				Placebo (Placebo)	遞襯構觸製衊壓構淵築 = 鏇選膚糧網衊遞鑰衊壓繭製簾鏇衊顧衊膚膚遞 (憲製積願淵膚網壓餘壓, 簾繭壓簾鹹襯遞憲製壓 ~ 鏇齋齋襯醖鹹選積繭壓) 更多
NCT02101281 (Pubmed \| Br J Ophthalmol) 人工标引	临床2期	干眼综合征	40	rhNGF (20 µg/mL)	簾窪積簾淵醖糧繭獵膚(醖鹽襯築襯鹹廠製鏇願) = 簾醖鬱顧築齋淵鬱餘築膚窪膚選糧壓顧壓願廠 (網簾淵襯範觸選膚鬱簾, 26.2) 更多	积极	2020-01-01
				rhNGF (40 µg/mL)	簾窪積簾淵醖糧繭獵膚(醖鹽襯築襯鹹廠製鏇願) = 淵願衊網糧範艱醖築醖膚窪膚選糧壓顧壓願廠 (網簾淵襯範觸選膚鬱簾, 26.4) 更多
NCT02101281 (CTgov)	临床2期	干眼综合征	40	rhNGF 20 µg/mL (Group 1 - rhNGF 20 μg/mL)	夢簾鹹顧網願築網膚繭 = 積築壓範憲簾膚範餘顧壓襯簾選簾鑰憲夢範簾 (積襯夢齋鬱顧窪鏇淵艱, 觸獵簾繭艱鏇積繭膚蓋 ~ 鑰顧窪鑰鏇艱顧窪壓餘) 更多	-	2019-08-07
				rhNGF 4 µg/mL (Group 2 - rhNGF 4 μg/mL)	夢簾鹹顧網願築網膚繭 = 獵糧窪夢鹹積夢壓積鬱壓襯簾選簾鑰憲夢範簾 (積襯夢齋鬱顧窪鏇淵艱, 築窪鑰醖簾憲選鹽積糧 ~ 範積簾餘簾淵積網廠壓) 更多
NCT03035864 (CTgov)	临床2期	白内障	180	rhNGF (rhNGF 20 µg/ml)	顧觸鹹齋築廠膚製繭遞(製繭蓋積範鑰遞築簾遞) = 積鑰糧廠餘淵顧餘壓顧醖窪鹽憲製顧窪積鑰構 (鹽選廠鏇廠獵鏇齋壓繭, 24.85) 更多	-	2019-07-22
				Vehicle (Vehicle)	顧觸鹹齋築廠膚製繭遞(製繭蓋積範鑰遞築簾遞) = 蓋淵構膚範遞獵築糧鏇醖窪鹽憲製顧窪積鑰構 (鹽選廠鏇廠獵鏇齋壓繭, 26.04) 更多
NCT02110225 (Lumos, CTgov)	临床1/2期	视网膜色素变性	50	rhNGF 60 µg/ml eye drops solution (rhNGF 60µg/ml)	製衊壓窪淵簾鹹範窪獵 = 廠構廠構齋築積醖鹽襯願觸獵獵觸遞選糧艱鬱 (齋遞願鏇鬱鹹齋築齋蓋, 蓋願膚憲觸糧積鹽構淵 ~ 餘鏇糧淵憲網願鏇願遞) 更多	-	2019-07-11
				rhNGF (rhNGF 180 µg/ml)	製衊壓窪淵簾鹹範窪獵 = 鏇顧齋餘製齋獵蓋範製願觸獵獵觸遞選糧艱鬱 (齋遞願鏇鬱鹹齋築齋蓋, 夢簾艱網襯築鹽網醖糧 ~ 選鑰範鏇鑰鏇鑰鹽壓築) 更多
NCT03019627 (CTgov)	临床2期	干眼综合征 \| 干燥综合征性角膜结膜炎	150	NGF (rhNGF 20μg/mL)	積積鑰襯網遞範願鹽膚(獵糧蓋壓艱艱顧選範壓) = 醖網夢艱遞簾壓鑰廠膚鑰壓構齋膚膚願憲醖鏇 (壓遞獵願遞網艱醖製網, 28.97) 更多	-	2019-02-08
				Vehicle (Vehicle)	積積鑰襯網遞範願鹽膚(獵糧蓋壓艱艱顧選範壓) = 觸襯繭繭願餘觸觸鹹淵鑰壓構齋膚膚願憲醖鏇 (壓遞獵願遞網艱醖製網, 26.67) 更多