A clinical evaluation of efficacy of Aaragwadhadi kashay in comparision with Deflazacort using EASI scale in Vicharchika with special reference to Atopic Dermatitis :a randomized control trial - NIL

开始日期2026-01-16

申办/合作机构-

CTRI/2025/12/099122

/ Not yet recruiting临床4期IIT

Balancing Efficacy and Safety: A prospective comparative study of Prednisolone and Deflazacort in adults with Steroid Responsive Dermatoses. - NIL

开始日期2025-12-24

申办/合作机构-

NCT07721610

/ Completed临床4期

Omega-3-Based Nutraceutical Supplementation as Adjunctive Therapy in Adults With Otitis Media With Effusion: A Multicenter, Double-Blind, Randomized Placebo-Controlled Trial

Brief Summary - OMENUTS Trial Otitis media with effusion (OME) is a condition in which fluid accumulates in the middle ear, causing conductive hearing loss, ear fullness, tinnitus, and Eustachian tube dysfunction. Although OME is well-recognized in children, it also affects adults, in whom it is frequently under-diagnosed and inadequately treated. No established, well-tolerated pharmacological therapy currently exists for adult OME, and current medical management options - including antihistamines, corticosteroids, and mucolytics - have shown inconsistent efficacy in clinical trials.
Omega-3 polyunsaturated fatty acids and associated nutraceutical compounds have well-documented anti-inflammatory and immunomodulatory properties that are mechanistically relevant to the pathophysiology of OME, which is driven by chronic low-grade mucosal inflammation, Eustachian tube dysfunction, and impaired mucociliary clearance. A nutraceutical combination containing omega-3 fatty acids, Spirulina, magnesium, and vitamin B6 has previously demonstrated efficacy in reducing inflammatory biomarkers and improving clinical outcomes in allergic rhinitis - a condition sharing key pathophysiological features with OME.
The OMENUTS trial is a multicenter, double-blind, randomized placebo-controlled trial designed to evaluate the efficacy of this nutraceutical combination as adjunctive therapy in adults with confirmed OME. The trial is conducted across three tertiary referral centres in Italy: Isola Tiberina - Gemelli Isola Hospital (Rome), the University of Catanzaro (Catanzaro), and Tortorella Clinic (Salerno).
A total of 501 adult participants aged 18 to 70 years with a confirmed diagnosis of OME - established by otoscopic examination and tympanometry (type B curve) of at least 4 weeks' duration - are randomized in a 1:1:1 ratio to one of three groups. All participants receive a shared background therapy of intranasal mometasone furoate spray and oral deflazacort for three weeks. In addition to the background therapy, participants in Group 1 (intervention group) receive the nutraceutical combination (omega-3 fatty acids, Spirulina, magnesium, and vitamin B6; one tablet daily) for three weeks; participants in Group 2 (active control group) receive oral bilastine 20 mg daily for three weeks; and participants in Group 3 (placebo group) receive an identical placebo tablet daily for three weeks.
The co-primary outcomes are tympanometric resolution - defined as normalization of the tympanometric curve from type B to type A - and audiometric improvement - defined as a reduction in pure-tone average of more than 15 dB HL - both assessed at the end of treatment (week 3, T1). Secondary outcomes include changes in patient-reported symptom severity (VAS), Eustachian tube dysfunction symptoms (ETDQ-7), psychosocial impact of hearing impairment (HHIA), and Eustachian tube patency assessed by the Valsalva maneuver. A 60-day post-treatment follow-up visit (T2) is included to evaluate recurrence rates across groups.
Randomization is computer-generated via REDCap, stratified by site, and implemented with permuted blocks of variable size. The trial is conducted under a double-blind design, with all investigational products identical in appearance, odour, and taste. Safety outcomes, including the incidence and nature of adverse events classified according to MedDRA criteria, are monitored continuously throughout the study.
The primary analysis is conducted on a modified intention-to-treat basis, including all participants who received at least one dose of the assigned treatment and completed at least one post-baseline assessment. The trial is conducted in accordance with the Declaration of Helsinki, CONSORT guidelines, and was approved by the Institutional Review Board of the Region of Calabria, Italy (approval code: 177/2026).

开始日期2025-10-01

申办/合作机构

GEMELLI ISOLA SOCIETA BENEFIT S.P.A.

100 项与地夫可特相关的临床结果

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100 项与地夫可特相关的转化医学

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100 项与地夫可特相关的专利（医药）

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721

项与地夫可特相关的文献（医药）

2026-08-01·FREE RADICAL BIOLOGY AND MEDICINE

Difenoconazole impairs male reproductive function by inducing spermatogenic ferroptosis via the Wnt/β-catenin/NCOA4-mediated ferritinophagy axis

Article

作者: Li, Dinggang ; Zheng, Xiangqin ; Wang, Siyuan ; Wei, Guanghui ; Wang, Runchang ; Zhao, Peng ; Deng, Wei ; Long, Chunlan ; Guo, Qitong ; Huang, Xu ; Liu, Xinyi ; Wu, Shengde ; Wei, Yuexin ; Shen, Lianju

Difenoconazole (DFZ), a widely used triazole fungicide, may pose a potential risk to male reproductive health, although its mechanistic basis in mammals remains incompletely understood. This study assessed DFZ-induced testicular injury through in vivo mouse models and in vitro GC-1 spermatogonia and GC-2 spermatocyte cell systems, conducting transcriptomic, metabolomic, and functional analyses concurrently. Chronic DFZ exposure was associated with dose-dependent testicular atrophy, disorganization of seminiferous tubule architecture, reduced sperm count, increased sperm malformation, and decreased serum testosterone levels, along with detectable DFZ accumulation in serum. Multi-omics profiling revealed coordinated molecular alterations in testicular tissue and germ cells, with ferroptosis-related pathways and lipid metabolic reprogramming being prominently enriched. In testicular tissue and germ cells, DFZ induced features consistent with ferroptosis, including depletion of GSH, accumulation of MDA and Fe²⁺, inactivation of GPX4, and mitochondrial damage. These changes were alleviated by Fer-1 and DFO, suggesting that ferroptosis contributes to DFZ-induced cytotoxicity. Moreover, GPX4 knockdown further aggravated DFZ-induced ferroptotic injury, supporting a central role for GPX4 in this process. Mechanistically, DFZ appeared to activate autophagy and NCOA4-mediated ferritinophagy, which may promote iron overload and lipid peroxidation. Wnt/β-catenin signaling activation reduced NCOA4 upregulation and ferroptotic damage, but this protective effect was nullified by NCOA4 silencing, suggesting NCOA4 operates downstream of Wnt/β-catenin signaling. These findings reveal that DFZ triggers spermatogenic ferroptosis through Wnt/β-catenin-NCOA4-mediated ferritinophagy, offering insights into triazole fungicide-induced male reproductive toxicity and highlighting potential targets for environmental infertility interventions.

2026-07-14·NEUROLOGY

Vamorolone for Duchenne Muscular Dystrophy

Article

作者: Bushby, Katherine ; Lampa, Erik ; Berglund, Anders ; Ward, Leanne M. ; Rochford, Edward Thomas James ; Guglieri, Michela ; Clemens, Paula R. ; McDermott, Michael P. ; Griggs, Robert C. ; Hoffman, Eric P. ; Schiava, Marianela ; James, Meredith K.

BACKGROUND AND OBJECTIVES:

Vamorolone demonstrated similar efficacy for Duchenne muscular dystrophy (DMD) compared with prednisone in a 24-week exploratory analysis and may reduce key side effects compared with classic corticosteroids. In this study, we compare the efficacy and anthropometric effect of vamorolone 6 mg/kg/d with prednisone 0.75 mg/kg/d and deflazacort 0.9 mg/kg/d in steroid-naïve boys aged 4 to <7 years using data from 2 trials.

METHODS:

VISION-DMD was a phase 2b, 48-week randomized, double-blind trial assessing vamorolone 2 and 6 mg/kg/d vs placebo and prednisone 0.75 mg/kg/d to 24 weeks. Finding the Optimum Steroid Regimen for DMD (FOR-DMD) was a double-blind, parallel-group randomized trial comparing daily prednisone 0.75 mg/kg/d, daily deflazacort 0.9 mg/kg/d, and intermittent prednisone 0.75 mg/kg (10/10 days on/off). Entropy balancing generated weighted data for mixed model for repeated measures analyses that compared VISION-DMD vamorolone 6 mg/kg/d efficacy and anthropometric outcomes with FOR-DMD prednisone and deflazacort outcomes at 6 and 12 months. Inclusion criteria were boys with genetically confirmed DMD, age 4 to <7 years, ambulatory, and able to complete a time-to-stand (TTSTAND) assessment in <10 seconds at baseline.

RESULTS:

All interventions showed motor improvements relative to baseline at 6 and 12 months. Using the weighted cohorts and at 12 months, vamorolone 6 mg/kg/d (n = 28) had similar changes in TTSTAND velocity, the primary motor endpoint component in both trials, vs daily prednisone (n = 50) or deflazacort (n = 55; mean baseline age 5.42 years all groups), but with CIs overlapping minimal clinically important thresholds of 0.023 rises per second (TTSTAND velocity least squares mean [LSM] difference [95% CI]: vamorolone 6 mg/kg/d vs prednisone 0.75 mg/kg/d, 0.004 rises per second [-0.025 to 0.032] and vs deflazacort 0.9 mg/kg/d, 0.001 rises per second [-0.027 to 0.028]). Body mass index (BMI) Z-scores increased in all groups, greatest with vamorolone (12-month LSM difference [95% CI]: vamorolone vs prednisone 0.57 [0.24-0.90]; vamorolone vs deflazacort 0.29 [0.03-0.56]), whereas growth slowdown occurred in prednisone- and deflazacort-treated boys but not vamorolone (12-month LSM difference in height Z-scores [95% CI]: vamorolone vs prednisone 0.57 [0.24-0.90]; vamorolone vs deflazacort 0.72 [0.53-0.91]).

DISCUSSION:

Vamorolone demonstrated numerically similar TTSTAND velocity changes to prednisone and deflazacort at 1 year; however, interpretations of differences are limited by 95% CIs crossing minimally important difference thresholds. Further evidence of the growth-protective effect of vamorolone was observed; however, all treatments increased BMI. Vamorolone provides a linear growth-protective classic corticosteroid alternative.

TRIAL REGISTRATION INFORMATION:

NCT03439670; NCT01603407.

CLASSIFICATION OF EVIDENCE:

This Class III study did not definitively identify differences in efficacy between vamorolone and classic corticosteroids but found that vamorolone protects linear growth in boys with DMD.

2026-07-01·Drug Testing and Analysis

Elimination Profile of Deflazacort and Metabolites in Urine and Plasma Following Oral Administration: Relevance to Sports Drug Testing

Article

作者: Ventura, Rosa ; Aldea‐Perona, Ana ; Carbó, Marcel·lí ; Coll, Sergi ; Monfort, Núria ; Bressan, Claudia

ABSTRACT:

Deflazacort (DEF) is a glucocorticoid prohibited in sports competitions when orally administered. This study aimed to assess the urinary and plasma excretion profiles of DEF and its metabolites after a single oral administration to verify the World Anti‐Doping Agency's minimum reporting level (MRL) of 30 ng/mL to detect administration of DEF. DEF was orally administered to eight healthy male participants (30 mg). Urine and plasma samples were collected before and after administration, and DEF and its metabolites were quantified using liquid chromatography–tandem mass spectrometry.Urinary concentrations confirmed that DEF is rapidly metabolized to 21‐desacetyl‐deflazacort (DES), with minimal detection of unchanged DEF. DES and the 6‐hydroxy metabolites, 6βOHDES and 6αOHDES, were present in most urine samples during the first 48 h post‐administration. DES peaked early (0–4 h) and declined below 30 ng/mL after 24 h, while 6βOHDES peaked later (4–8 h) and remained above 30 ng/mL until 36–48 h. 6αOHDES was detected at lower concentrations and cleared rapidly.In plasma, DES and 6βOHDES were detected within 8 h, with DES showing higher peak levels and longer half‐life. DEF itself was not detected in plasma. Pharmacokinetic modeling showed good agreement between urinary and plasma excretion data. Cortisol levels were suppressed post‐dose, indicating systemic GC activity, but recovered by 72 h in most subjects. The findings support the use of DES as the primary urinary marker for DEF detection, confirm the suitability of the MRL of 30 ng/mL and support the recommendation of a 3‐day washout period due to interindividual variability.

242

项与地夫可特相关的新闻（医药）

2026-08-05

·终身学习-星辰如梦

【药品分享】阿柏西普眼内注射溶液——重组人源VEGF融合蛋白的治疗用生物制品

阿柏西普眼内注射溶液（艾力雅®，Eylea）是Bayer AG（拜耳）研发的一款重组人源VEGF融合蛋白的治疗用生物制品，仅用于玻璃体腔眼内注射给药。基本信息作用机制：可同时结合VEGF‑A、VEGF‑B、PlGF（胎盘生长因子），抑制眼底异常新生血管，降低血管渗漏，减轻黄斑水肿；靶点覆盖多于单纯抗VEGF单抗类眼科药物。国内获批适应症（原研艾力雅）：新生血管（湿性）年龄相关性黄斑变性 (nAMD)；糖尿病性黄斑水肿 (DME)；视网膜静脉阻塞继发黄斑水肿 (RVO‑ME)；糖尿病视网膜病变（DR）。国内获批规格与用法用量 ✅2mg规格（40 mg/mL）：单次玻璃体腔注射给药2 mg（50 μL） nAMD：前3针每月1次，之后每8周给药1次； DME/RVO‑ME：前5针每月1次，后续按需给药。 ✅8mg高剂量规格（114.3 mg/mL，艾力雅）国内获批适应症：湿性年龄相关性黄斑变性（nAMD）给药方案：初始连续3个月每月玻璃体腔注射8 mg；之后依据视力、眼底解剖评估，最长可每4个月（16周）给药一次，大幅降低注射频次。关键Ⅲ期PULSAR研究：96周，8 mg组平均注射8.2次，对比2 mg组少约 4 次注射，视力获益等效，未新增显著安全风险信号。给药方式：必须由具备玻璃体腔注射资质的眼科医生操作。主要风险：眼内炎、视网膜脱离、眼压升高、玻璃体出血等注射相关眼部不良反应。上市情况国外上市信息 2 mg原研拜耳（Eylea/艾力雅®）：FDA 2011‑11获批上市，全球多国上市，适应症覆盖nAMD、DME、RVO‑ME、DR、近视性CNV、早产儿视网膜病变等。 8mg 高剂量版 Eylea FDA：2023‑08获批，后续陆续批准nAMD、DME、DR、RVO‑ME；2026‑04 美国批准最长5个月给药间隔。欧盟：2024‑01 获批，适应症为nAMD、DME。中国上市信息 2 mg 规格（40 mg/mL）：NMPA 2018‑02获批，商品名艾力雅®。适应症为nAMD、DME、RVO‑ME、糖尿病视网膜病变（DR）。 8 mg 高剂量规格（114.3 mg/mL，艾力雅）：NMPA 2025‑05‑22获批，国内仅获批nAMD（湿性老年黄斑变性），DME/RVO‑ME暂未获批 8 mg适应症。国内生物类似药上市信息（均仅 2 mg规格，暂无 8mg）美国紫皮书信息注册受理信息临床试验信息专利情况原研专利核心化合物/序列专利制剂/处方/组合物专利用途/新适应症专利给药装置/给药方案/组合疗法相关专利基因/AAV/细胞/制备纯化相关专利国内申请人专利（非原研）其他药品分享链接如下，欢迎点击查看： Ormeloxifene OTF慢性体重管理的新型药物-Tirzepatide新型的非阿片类小分子镇痛药-Suzetrigine奥麦利昔芬口腔薄膜立项调研报告纳米技术双氯芬酸乳胶剂药学研究2025年9月药品批准信息快讯（八）——利斯的明透皮贴剂分享2025年9月药品批准信息快讯（九）——米托坦片分享CDE-《化学仿制药参比制剂目录（第一百批）》（征求意见稿）[附: 药品分享——阿达苏(洛沙平吸入剂)]药品分享-塞来昔布盐酸曲马多片药品分享——伐莫洛龙口服混悬液【药品分享】盐酸菲优拉生片【药品分享】拉坦噻吗滴眼液【药品分享】Baxdrostat 片【药品分享】Omecamtiv Mecarbil缓释片【药品分享】曲地匹坦（Rolapitant）【药品分享】Zasocitinib 胶囊【药品分享】甲磺酸阿帕替尼片【药品分享】巴氯芬口服溶液【药品分享】奥德昔巴特胶囊【药品分享】扎维吉泮鼻喷雾剂【药品分享】左羟丙哌嗪糖浆【药品分享】利丙双卡因凝胶贴膏【药品分享】司来吉兰改良型新药（缓释片）【药品分享】复方甘菊利多卡因凝胶【药品分享】盐酸索安非托片【药品分享】地塞米松口溶膜【药品分享】盐酸来罗西利片【药品分享】马来酸噻吗洛尔凝胶【药品分享】褪黑素颗粒【药品分享】罗氟司特乳膏【药品分享】盐酸芬戈莫德胶囊【药品分享】示踪用盐酸米托蒽醌注射液【药品分享】美洛昔康纳米晶注射液【药品分享】Clascoterone 5%外用溶液【药品分享】马来酸依那普利口服溶液【药品分享】注射用双氯芬酸钠利多卡因【药品分享】马来酸依那普利叶酸片—复方降压药【药品分享】依曲帕米鼻喷雾剂—用于治疗成人阵发性室上性心动过速的可自行给药鼻喷雾剂【药品分享】奥卡西平口服混悬液—一款可用于儿童的钠通道调节类抗癫痫药【药品分享】泊沙康唑口服混悬液——一种常用的抗真菌药物【药品分享】舒沃替尼片【药品分享】依伏卡塞片——第二代口服拟钙剂【药品分享】西诺氨酯片——第三代抗癫痫发作药物【药品分享】匹妥布替尼片——非共价可逆 BTK 抑制剂（用于复发 / 难治套细胞淋巴瘤（MCL））【药品分享】贝美前列素【产品分享】达普司他片【药品分享】水合氯醛糖浆——镇静催眠药【产品分享】苏沃雷生（Suvorexant）——全球首个食欲素受体拮抗剂类催眠药【药品分享】贝沙罗汀【药品分享】地夫可特干混悬剂——一种糖皮质激素类药物【药品分享】伊卢多啉片——治疗成人腹泻型肠易激综合征（IBS‑D）的首创口服阿片受体调节剂【药品分享】卢美哌隆胶囊——一种新型的非典型抗精神病药物【药品分享】奥氟格列隆片——口服非肽类小分子 GLP-1 受体激动剂【药品分享】苯磺酸克利加巴林胶囊——一种新型的治疗神经病理性疼痛的药物【药品分享】苯磺酸美洛加巴林片——治疗周围神经病理性疼痛（PNP）的第三代钙离子通道调节剂【药品分享】Iberdomide胶囊——一款新一代cereblon（CRBN）E3连接酶调节剂（CELMoD）化合物【药品分享】酒石酸伐尼克兰鼻喷雾剂——一种用于治疗干眼症的西药【药品分享】赛沃替尼片——中国首个获批的高选择性 MET 酪氨酸激酶抑制剂【药品分享】盐酸阿夫唑嗪缓释片——一种用于治疗良性前列腺增生（BPH）的 α1 - 受体阻滞剂【药品分享】索格列净片——全球首个获批的SGLT1/SGLT2 双重抑制剂【药品分享】奥吡卡朋胶囊——新一代的儿茶酚-氧位-甲基转移酶抑制剂（COMT）【药品分享】奎扎替尼片——一种口服高选择性 FLT3 抑制剂【药品分享】硫酸拉罗替尼胶囊 / 口服溶液——全球首个高选择性口服 TRK 抑制剂【药品分享】司帕生坦片——成人IgA肾病领域首个非免疫抑制疗法【药品分享】黄体酮阴道栓剂/阴道缓释凝胶剂——主要用于辅助生殖技术（ART）中的黄体支持【药品分享】替戈拉生片——首款国产钾离子竞争性酸阻滞剂类药物（P-CAB）【药品分享】拉坦前列烯酯滴眼剂——一种新型眼科用药【药品分享】布比卡因脂质体注射液——一种长效局部麻醉药【药品分享】甲磺酸加诺沙星片——一种喹诺酮类抗菌药【药品分享】磷酸芦可替尼乳膏——中国首款且唯一获批的白癜风治疗靶向药【药品分享】甲磺酸洛美他派胶囊——全球唯一口服MTP抑制剂【药品分享】依达拉奉口服混悬液——肌萎缩侧索硬化症（ALS，渐冻人症）新药【药品分享】依达拉奉右莰醇舌下片——全球卒中领域首个获FDA突破性疗法认定的舌下脑保护剂【药品分享】马来酸氟诺替尼片——JAK2/FLT3/CDK6 三靶点抑制剂【药品分享】普拉替尼胶囊——国内首个获批的高选择性 RET（转染重排）酪氨酸激酶抑制剂【药品分享】右美托咪定舌下膜——新型 α₂肾上腺素受体激动剂舌下膜剂【药品分享】卡匹色替片——全球首个获批上市的AKT抑制剂【药品分享】库莫西利胶囊——全球首个CDK2/4/6抑制剂【药品分享】赛贝曲司他片——全球首个HAE急性发作口服疗法【药品分享】KYGEVVI（Doxecitine/Doxribtimine）——全球首款且唯一获批的TK2d针对性治疗药物【药品分享】Forzinity（Elamipretide）注射剂——全球首个用于治疗Barth综合征的药物【药品分享】Brinsupri（Brensocatib, 布伦索卡替布）片——全球首个获批上市的二肽基肽酶1（DPP-1）抑制剂【药品分享】维立西呱片——全球首个心衰适应症的可溶性鸟苷酸环化酶（sGC）刺激剂【药品分享】盐酸哌罗匹隆片——非典型抗精神病药【药品分享】盐酸托泊替康胶囊/注射用盐酸托泊替康——经典的拓扑异构酶Ⅰ抑制剂类抗肿瘤药【药品分享】阿普昔腾坦片——全球首个获批用于难治性高血压的口服双重内皮素受体拮抗剂（ERA）【药品分享】注射用盐酸依拉环素——全球首个氟环素类抗生素【药品分享】奥洛格列净胶囊——国内首款抑制SGLT1与SGLT2的1类创新药【药品分享】盐酸匹米替尼胶囊——国内首个、全球首个获批的腱鞘巨细胞瘤（TGCT）系统性靶向药，高选择性CSF-1R抑制剂【药品分享】布地奈德肠溶胶囊——全球首个、中国唯一获批用于原发性IgA肾病对因治疗的靶向药物【药品分享】艾拉莫德片——中国原研、全球首个上市的小分子抗风湿药（csDMARD）【药品分享】索托克拉片——新一代BCL2抑制剂【药品分享】利奈昔巴特片——口服、选择性、可逆性回肠胆汁酸转运体（IBAT/ASBT）抑制剂【药品分享】多替诺雷片——高选择性URAT1抑制剂【药品分享】瑞普泊肽片——口服GLP‑1/GIP双靶点激动剂【药品分享】硫酸索西美雷塞片——口服、强效的KRAS G12C共价抑制剂【药品分享】罗伐昔替尼片——全球首创JAK/ROCK双靶点口服小分子抑制剂【药品分享】埃诺格鲁肽注射液——全球首个获批上市的cAMP偏向型长效GLP-1受体激动剂【药品分享】维培那肽注射液——长效GLP-1受体激动剂【药品分享】普乐司兰钠注射液——全球首个靶向APOC3 mRNA的siRNA降脂药【药品分享】玛仕度肽注射液——全球首个GCG/GLP-1双激动剂【药品分享】瑞米布替尼片——全球首个获批用于CSU的口服靶向药【药品分享】溴莫尼定噻吗洛尔滴眼液——α₂受体激动剂与非选择性 β 受体阻滞剂组成的复方降眼压药【药品分享】吡洛西利片——国内首个CDK2/4/6多靶点抑制剂【药品分享】奥氟格列隆片（Orforglipron） ——中国首个、全球首批上市的口服小分子 GLP-1受体激动剂【药品分享】Copper Histidine注射液（Zycubo）——全球首个、唯一用于治疗儿童门克斯病（Menkes disease）的铜替代注射剂【药品分享】依来格列隆片（Elecoglipron）——口服小分子非肽类GLP-1受体激动剂【药品分享】阿利奈普仑（Aleniglipron）——口服小分子、非肽类、偏向性GLP-1受体激动剂【药品分享】甲磺酸阿美替尼片——中国首个原研三代EGFR酪氨酸激酶抑制剂（TKI）【药品分享】甲磺酸达麦利替尼片——口服高选择性c-MET酪氨酸激酶抑制剂【药品分享】盐酸Acoramidis片——转甲状腺素蛋白（TTR）稳定剂【药品分享】阿莫西林伏诺拉生胃漂浮片——3D打印胃漂浮片【药品分享】盐酸伊可白滞素片——全球首创口服IL‑23受体拮抗剂【药品分享】马来酸吡咯替尼片——中国首个自研的HER1/HER2/HER4不可逆酪氨酸激酶抑制剂（TKI）【药品分享】本维莫德乳膏——全球首创芳香烃受体（AhR）调节剂（TAMA）类皮肤病外用药【药品分享】盐酸莫托咪酯注射液——化药1类静脉麻醉新药【药品分享】注射用罗哌卡因微晶——长效缓释局部麻醉药【药品分享】盐酸伊立替康脂质体注射液——一款针对吉西他滨治疗失败的转移性胰腺癌的二线标准脂质体化疗药【药品分享】甲磺酸氟马替尼片——Bcr-Abl酪氨酸激酶选择性抑制剂【药品分享】注射用阿加糖酶β——国内获批的首个用于治疗法布雷病的药物【药品分享】甲苯磺酸尼拉帕利胶囊——高选择性PARP1/2抑制剂【药品分享】帕米帕利胶囊——强效、选择性PARP1/2抑制剂【药品分享】苯环喹溴铵鼻喷雾剂——选择性M胆碱能受体拮抗剂【药品分享】注射用拉罗尼酶浓溶液——MPS Ⅰ型的特异性酶替代治疗药物【药品分享】海博麦布片——国产自主原研的胆固醇吸收抑制剂【药品分享】盐酸可洛派韦胶囊——直接抗病毒药物（DAA）→ NS5A抑制剂【药品分享】克拉考特酮乳膏——外用、非甾体、选择性雄激素受体（AR）抑制剂【药品分享】非卢替尼片——全球首个用于多发性硬化的口服BTK抑制剂【药品分享】Brenipatide注射液——GLP-1R/GIPR双受体激动剂（继替尔泊肽之后的第二款）【药品分享】罗赛促红素α注射液——国产首个长效重组促红细胞生成素（EPO）创新药【药品分享】贝组替凡片——全球首个口服小分子HIF-2α抑制剂【药品分享】那米司特片——口服选择性PDE4B抑制剂【药品分享】富马酸立康可泮胶囊——一款针对罕见病PNH的口服补体B因子抑制剂【药品分享】醋酸索乐匹尼布片——高选择性脾酪氨酸激酶（Syk）抑制剂【药品分享】甲磺酸艾多替尼片——全球首款专为肺癌脑转移设计的第三代EGFR-TKI 【药品分享】安瑞克芬注射液——全球首个获批镇痛适应症的高选择性外周κ-阿片受体（KOR）激动剂【药品分享】Difelikefalin注射液——全球首个获批的外周选择性κ-阿片受体（KOR）激动剂【药品分享】Obefazimod——口服、靶向 CBC、特异性增强miR-124的首创小分子【药品分享】拉尼兰诺片——口服泛PPAR激动剂【药品分享】舒洛地特注射液——抗血栓药、血管保护剂【药品分享】贝普若韦生注射液——反义寡核苷酸（ASO）类乙肝新药【药品分享】氨酚右敏口服溶液（奥肯能®）——中国内地唯一获批的氨酚右敏口服溶液【药品分享】佩玛贝特片——新型过氧化物酶体增殖物激活受体α/δ双重激动剂 (PPARα/δ) 【药品分享】盐酸依匹斯汀乳膏——外用第二代非镇静抗组胺抗过敏药膏【药品分享】注射用双羟萘酸帕瑞肽微球——一款用于治疗肢端肥大症的第二代长效生长抑素类似物（SRL）微球制剂【药品分享】埃拉菲布拉诺片——治疗原发性胆汁性胆管炎（PBC）的口服靶向新药【药品分享】泽卢克布仑钠注射液——新一代皮下注射型C5补体抑制剂【药品分享】依达拉奉右莰醇注射用浓溶液——一款复方神经保护类注射剂【药品分享】醋酸戈那瑞林注射液——促性腺激素释放激素（GnRH）类药物【药品分享】甲磺酸贝舒地尔片——全球首个选择性ROCK2激酶抑制剂【药品分享】伊托法替布软膏——外用JAK抑制剂（JAK1/JAK3）【药品分享】注射用华卟啉钠——新型肿瘤光动力治疗（PDT）专用光敏剂【药品分享】优替德隆胶囊——口服埃博霉素类微管抑制剂【药品分享】艾普美妥司他片——EZH1/2双靶创新药【药品分享】精氨酸艾曲莫德片——新型选择性鞘氨醇1-磷酸受体（S1P₁）调节剂【药品分享】磷酸芦可替尼乳膏——全球/国内首款、唯一获批用于非节段型白癜风复色的外用JAK1/JAK2选择性抑制剂【药品分享】盐酸右哌甲酯缓释胶囊——治疗6岁及以上注意缺陷多动障碍（ADHD）的一线药物【药品分享】抗菌肽PL-3301口腔凝胶——中国首创的抗菌肽类抗口腔真菌感染药物【药品分享】Zasocitinib (TAK-279) 胶囊——新一代高选择性口服 TYK2 抑制剂【药品分享】注射用Abelacimab——一款全人源IgG1单克隆抗体，靶向凝血因子XI（FXI）【药品分享】Duvakitug注射液——首创性人源化IgG1-λ2单克隆抗体，靶向TL1A（TNFSF15）【药品分享】醋酸氟轻松/氢醌/维A酸乳膏——治疗中重度黄褐斑疗效明确的外用复方【药品分享】富马酸伏诺拉生氯化钠注射液——钾离子竞争性酸阻滞剂（P‑CAB）||东阳光药业研发的2.2,2.4类改良新型新药【药品分享】盐酸阿曲生坦片——诺华研发的高选择性内皮素 A（ETA）受体拮抗剂||中国首个获批用于IgA 肾病的非免疫性口服靶向药【药品分享】RGT-274-CR胶囊——上海齐鲁锐格医药研发的口服小分子GLP-1受体激动剂【药品分享】酒石酸西尼必利片——高选择性5-HT₄受体激动剂类胃肠动力药【药品分享】石杉碱甲控释片——采用双相控释/渗透泵技术的2.2类改良型新药【药品分享】塞伐艾替尼片——口服、可逆、选择性酪氨酸激酶抑制剂（TKI）【药品分享】Admilparant片——口服小分子LPA₁受体拮抗剂【药品分享】瑞拉可兰胶囊——全球首个选择性糖皮质激素受体（GR）拮抗剂【药品分享】盐酸依特卡肽注射液——全球首个、国内唯一获批的静脉注射型拟钙剂【药品分享】Avacincaptad pegol玻璃体内注射液——全球首批用于AMD继发地图样萎缩（GA）的靶向药物【药品分享】盐酸达利雷生片——新一代非管制、非苯二氮䓬类抗失眠药物【药品分享】甲磺酸瑞索利塞片——高选择性PI3Kα小分子靶向抑制剂 || 全球首个获批用于特定卵巢癌的靶向新药【药品分享】富马酸西他沙星注射液——新型喹诺酮类抗菌药物【药品分享】Icotrokinra——全球首个获批的口服 IL-23 受体（IL-23R）选择性拮抗多肽【药品分享】氘可来昔替尼片（Deucravacitinib）——全球首个口服、选择性 TYK2 变构抑制剂【药品分享】柯美奇拜单抗注射液——鼻用糖皮质激素治疗后症状控制不佳的成人中重度季节性过敏性鼻炎患者的福音【药品分享】吉诺昔替尼——口服小分子JAK1/TYK2双重抑制剂【药品分享】洛替拉纳（Lotilaner）——GABA 门控氯离子通道非竞争性拮抗剂 || 兼具兽医驱虫与人用眼科两大用途【药品分享】CagriSema——由卡格列肽（cagrilintide）2.4 mg与司美格鲁肽（semaglutide）2.4 mg组成的强效减重复方药【药品分享】苹果酸卡博替尼——口服、多靶点酪氨酸激酶抑制剂（TKI）【药品分享】迪高替尼（Delgocitinib）——一款外用泛JAK抑制剂【药品分享】拉氟莫司(Laflunimus)——口服免疫抑制剂 || 兼具免疫抑制与抗炎双重活性【药品分享】伏昔尼布片——全球首个IDH1/IDH2双重突变抑制剂靶向药【药品分享】盐酸奥昔布宁片——M受体抗胆碱药【药品分享】贝泽昔替尼片——国内首款获批的高选择性JAK2单靶点抑制剂【药品分享】苯甲酸安达艾替尼胶囊——新型小分子酪氨酸激酶不可逆抑制剂（EGFR-TKI）【药品分享】伏欣奇拜单抗注射液——全人源抗IL‑1β单克隆抗体 || 用于急性痛风性关节炎的1类创新生物药【药品分享】利多卡因丁卡因乳膏【药品分享】伐米奇拜单抗注射液——玻璃体内注射用抗IL-6人源化单抗【药品分享】依赛弗酶α注射液（Efzimfotase alfa 注射液）——第二代重组组织非特异性碱性磷酸酶（TNSALP）酶替代疗法【药品分享】格达托利西布（Gedatolisib）——first‑in-class 泛 PI3K/mTORC1/2 双重抑制剂【药品分享】伯瑞替尼肠溶胶囊——中国原研、高选择性c-MET小分子抑制剂 || 首个获批MET扩增NSCLC单药治疗的靶向药【药品分享】力胜克拉片——中国首个申报上市的原研Bcl-2抑制剂、全球第二款Bcl-2抑制剂【药品分享】BGM0504注射液——GLP-1/GIP 双受体激动剂【药品分享】塞利尼索片——全球首个口服选择性核输出蛋白XPO1抑制剂【药品分享】Seltorexant薄膜包衣片——选择性食欲素2受体（OX2R）高选择性拮抗剂【药品分享】盐酸埃克替尼片——国内首个自主研发的一代EGFR-TKI 【药品分享】盐酸伊普可泮胶囊——全球首个口服补体B因子抑制剂【药品分享】恩考芬尼胶囊——靶向BRAF V600突变的口服激酶抑制剂【药品分享】Enpatoran——口服、选择性TLR7/8小分子抑制剂【药品分享】硝酸异康唑阴道片——抗真菌外用药物【药品分享】Zorevunersen注射液——全球独家ASO上调蛋白表达技术【药品分享】妥拉美替尼胶囊——全球首个针对NRAS突变黑色素瘤的MEK抑制剂【药品分享】盐酸替那帕诺片——全球首创NHE3抑制剂类新型降磷药物【药品分享】注射用莱古比星——新型蒽环类抗肿瘤药【药品分享】优替德隆胶囊——口服埃博霉素类微管抑制剂【药品分享】伊那利塞片——中国首个PIK3CA突变乳腺癌精准靶向药【药品分享】呋喹替尼胶囊——中国首个自主研发VEGFR-TKI || VEGFR靶点靶向抗癌药【药品分享】琥珀酸瑞波西利片——CDK4/6抑制剂【药品分享】盐酸替那帕诺片——全球首个且唯一获批的肠道磷吸收抑制剂【药品分享】芬布替尼片——口服、可逆、可透过血脑屏障的BTK抑制剂【药品分享】炔丙基半胱氨酸胶囊——全球首个进入临床的硫化氢（H₂S）供体I类创新药【药品分享】Balcinrenone达格列净胶囊——选择性盐皮质激素受体拮抗剂（MRA）+SGLT-2抑制剂【药品分享】诺拉西班（Nolasiban）分散片——选择性口服催产素受体拮抗剂 || 早产二线/辅助治疗药物【药品分享】塞多明基注射液——国内首款裸质粒基因治疗药物【药品分享】鲁兹诺雷钠片——国产首个高选择性URAT1抑制剂【药品分享】玛伐凯泰胶囊——全球首创、中国唯一获批的心肌肌球蛋白抑制剂【药品分享】依托芬那酯凝胶——外用非甾体抗炎镇痛药【药品分享】聚卡波非钙片——国际指南首选容积性缓泻剂【药品分享】Mezigdomide胶囊——新一代IMiDs/cereblon E3泛素连接酶调节【药品分享】洛布替尼片——全球首款共价+非共价双机制第四代 BTK 靶向药【药品分享】奥莱泊肽注射液——PEG修饰长效GLP‑1单靶点激动剂【药品分享】安瑞曲替尼胶囊 ——国内第二款国产二代NTRK广谱抗癌药【药品分享】麦格司他胶囊——抑制葡萄糖神经酰胺合成酶的罕见病口服靶向药【药品分享】乌帕替尼片（Upadacitinib）——选择性JAK1抑制剂【药品分享】Dordaviprone胶囊——全球首款、目前唯一获批用于 H3 K27M 突变型弥漫性中线胶质瘤（DMG）的靶向药【药品分享】屈昔多巴胶囊——去甲肾上腺素前体药物 || 帕金森、多系统萎缩、神经源性体位低血压一线对症用药【药品分享】康特替尼颗粒——第二代ALK多靶点激酶抑制剂【药品分享】盐酸兰诺可泮片——国产首个获批上市的补体因子B（CFB）抑制剂【药品分享】Elinzanetant软胶囊——全球首款NK1/NK3双重神经激肽受体拮抗剂【药品分享】Fosmanogepix注射液——全球首创（First-in-class）Gwt1抑制剂类抗真菌前药【药品分享】Zorevunersen注射液——反义寡核苷酸药物【药品分享】宗艾替尼片——高选择性不可逆HER2（ERBB2）酪氨酸激酶抑制剂（TKI）【药品分享】甲苯磺酸他拉唑帕利胶囊——高选择性PARP抑制剂【药品分享】Bexicaserin口服溶液——选择性5-HT2C受体激动剂口服液体制剂【药品分享】醋酸伏诺拉生注射用浓溶液——钾离子竞争性酸阻滞剂（P-CAB）【药品分享】甲苯磺酸依仑司群片——新一代口服选择性雌激素受体降解剂（SERD）【药品分享】硫酸瑞美吉泮口崩片——CGRP 受体拮抗剂（降钙素基因相关肽）|| 偏头痛靶向特效药【药品分享】阿托吉泮片——全球首款口服长效CGRP靶点预防偏头痛药物【药品分享】乌布吉泮片——CGRP受体拮抗剂 || 第二代口服偏头痛急性期药物【药品分享】扎维吉泮鼻喷雾剂——高选择性CGRP受体拮抗剂 || 全球唯一获批鼻喷CGRP吉泮类急性期药物【药品分享】培来加南喷雾剂——全球首款上市加南类多肽抗菌药（FIC）【药品分享】艾沙利酮片——高选择性非甾体醛固酮受体拮抗剂（MRA） || 全球首个上市的非甾体选择性MR拮抗剂【药品分享】布美他尼鼻喷雾剂——全球首款、唯一鼻内给药袢利尿剂【药品分享】注射用盐酸依拉环素——新型全合成氟环素类（四环素衍生物）静脉抗生素【药品分享】舒瑞基奥仑赛注射液——全球首款实体瘤自体CLDN18.2靶向CAR-T细胞新药【药品分享】盐酸环喷托酯滴眼液——儿童验光首选短效睫状肌麻痹剂之一【药品分享】盐酸赛庚啶口服溶液——抗过敏抗组胺药【药品分享】Rinzimetostat (ORIC-944)——口服选择性 EED别构型PRC2表观遗传抑制剂【药品分享】Empasiprubart注射用浓溶液——全球首创靶向补体C2人源化IgG1清扫型单克隆抗体【药品分享】阿更葡糖钠注射液——全球第二款、中国首款自主研发的环糊精类靶向肌松逆转剂【药品分享】盐酸罗哌卡因缓释溶液——国产自主长效局麻缓释制剂 || 填补国内切口原位长效镇痛空白【药品分享】Telisotuzumab Adizutecan 注射用粉末——抗c-Met抗体偶联药物（ADC）【药品分享】L606 (曲前列尼尔脂质体吸入混悬液)——靶向肺动脉高压【药品分享】佩米替尼片——选择性FGFR1/2/3酪氨酸激酶抑制剂【药品分享】唑尼沙胺胶囊——磺酰胺类结构 || 广谱抗癫痫药，偏头痛预防【药品分享】琥珀酸安维吖啶片——双靶点胆碱酯酶抑制剂【药品分享】布莱昔替尼片（Bleximenib）——口服高选择性Menin-KMT2A相互作用抑制剂【药品分享】利厄替尼片——第三代EGFR-TKI靶向药【药品分享】注射用苯磺酸瑞马唑仑——苯二氮䓬类短效静脉镇静/麻醉药【药品分享】瑞维鲁胺片——国产全新二代雄激素受体抑制剂【药品分享】塞普替尼胶囊——全球首款高选择性RET靶向药 || 国内唯一拥有12岁以上儿童甲状腺肿瘤完整临床证据的RET抑制剂【药品分享】注射用和厚朴酚脂质体冻干粉——全球首创小分子脂质体靶向制剂【药品分享】注射用顺铂聚合物胶束——前沿纳米化疗创新赛道 || 改良型新药-纳米靶向注射制剂【药品分享】奥普雷顿片——国内首个获批的口服OX2R选择性激动剂【药品分享】琥珀酸思普可泮片——口服选择性补体B因子（CFB/FB）小分子抑制剂 || 全球首个每日一次口服FB抑制剂【药品分享】苹果酸法米替尼胶囊——多靶点TKI创新药 || 国内唯一获批用于宫颈癌的口服抗血管TKI 【药品分享】盐酸伊普可泮胶囊【药品分享】枸橼酸伊沙佐米胶囊——第二代、全球首个口服可逆性蛋白酶体抑制剂【药品分享】注射用西罗莫司（白蛋白结合型）——mTOR 激酶抑制剂 || 白蛋白纳米粒递送系统【药品分享】Bleximenib片——Menin抑制剂（KMT2A/Menin 相互作用抑制剂）【药品分享】Eloralintide注射液——选择性长效胰淀素（Amylin）受体激动剂（AMY1R激动剂）免责声明：本文仅作医药信息传播分享，并不构成投资或决策建议。

2026-08-03

·PRNewswire

PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2026 Financial Results

– Robust Q2 performance with total revenue of $361 million, including $239 million of product revenue – – Full-year 2026 expected total revenue increased to $1.18 to $1.28 billion, with full-year product revenue guidance raised to $850 to $950 million – – Sephience™ (sepiapterin) Q2 2026 revenue of $151 million, with continued broad uptake and geographical expansion – – Strong cash position of $2.2 billion as of June 30, 2026 – WARREN, N.J., July 30, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the second quarter ended June 30, 2026. "Our teams delivered another strong quarter enabling us to again raise 2026 product revenue guidance," said Matthew B. Klein, M.D., Chief Executive Officer. "We also made a number of advances in our R&D pipeline, including identification of our next small molecule splicing development candidate, PTC303, targeting somatic expansion disorders including Huntington's disease and myotonic dystrophy. In addition, we initiated the Phase 1 study of PTC612, our differentiated NLRP3 small molecule." Key Corporate Updates Q2 2026 total revenue of $361 million, including $239 million of product revenue Sephience global launch continues with strong momentum Q2 2026 revenue of $151 million, with the majority from the US and increasing contributions internationally 1,647 patients on commercial therapy worldwide as of June 30, 2026 Positive results from votoplam PIVOT-HD long-term extension study reported and enrollment in global Phase 3 INVEST-HD study ongoing PIVOT-HD 24-month results demonstrated dose-dependent disease slowing on cUHDRS scale in Stage 2 participants, reaching 52% at 10 mg dose level Plan to engage with FDA in 2H 2026 to discuss PIVOT-HD 24-month results being finalized INVEST-HD study being conducted and funded by partner Novartis with target enrollment of approximately 770 individuals with early symptomatic disease INVEST-HD initiation triggered a $50 million milestone payment from Novartis to PTC in Q2 2026 Vatiquinone PROVE-FA study to support NDA resubmission for treatment of Friedreich's ataxia expected to initiate in Q3 2026 Global study will enroll approximately 120 individuals with Friedreich's ataxia age 7 to 21 Study includes open-label treatment arm with matched natural history control group PTC pipeline continues to advance PTC303 named as MSH3 splicing program development candidate targeting Huntington's disease and myotonic dystrophy with plan to enter clinic in 2027 Phase 1 study of PTC612, an oral NLRP3 inhibitor, initiated in Q2 2026 Phase 2a study of PTC844, a next-generation DHODH inhibitor, expected to initiate in Q3 2026 Hege Sollie-Zetlmayer has been appointed to PTC's Board of Directors. Ms. Sollie-Zetlmayer has over 30 years of experience in the biopharmaceutical and medical device industry as a global business operations and human resources leader. She most recently served as PTC's Chief Human Resources Officer prior to her retirement in June 2026. Second Quarter 2026 Financial Highlights Total net product revenue and royalty revenue was $309.9 million for the second quarter of 2026, compared to $175.9 million for the second quarter of 2025. Total net product revenue across the commercial portfolio was $238.8 million for the second quarter of 2026, compared to $118.3 million for the second quarter of 2025, representing over 100% increase. Sephience net product revenues were $151.3 million for the second quarter of 2026, representing 21% growth compared to the first quarter of 2026. Translarna™ (ataluren) net product revenues were $42.2 million for the second quarter of 2026, compared to $59.5 million for the second quarter of 2025. Emflaza® (deflazacort) net product revenues were $24.6 million for the second quarter of 2026, compared to $36.4 million for the second quarter of 2025, due to continued generic erosion. Roche reported Evrysdi® (risdiplam) year-to-date sales of approximately 968 CHF million, resulting in royalty revenue of $71.1 million to PTC for the second quarter of 2026, compared to $57.6 million to PTC for the second quarter of 2025. In the second quarter of 2026, PTC recorded a development milestone of $50.0 million from Novartis for the first patient dosed in the ongoing INVEST-HD Phase 3 study. This sales milestone was recorded as collaboration revenue. Based on US GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $99.2 million for the second quarter of 2026, compared to $113.0 million for the second quarter of 2025. Non-GAAP R&D expenses were $88.6 million for the second quarter of 2026, excluding $10.5 million in non-cash, stock-based compensation expense, compared to $104.0 million for the second quarter of 2025, excluding $9.0 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $80.6 million for the second quarter of 2026, compared to $85.3 million for the second quarter of 2025. Non-GAAP SG&A expenses were $67.9 million for the second quarter of 2026, excluding $12.7 million in non-cash, stock-based compensation expense, compared to $75.7 million for the second quarter of 2025, excluding $9.5 million in non-cash, stock-based compensation expense. Net income was $83.5 million for the second quarter of 2026, compared to net loss of $64.8 million for the second quarter of 2025. In June 2026, PTC issued $550.0 million of senior convertible notes due in 2031 at 0% coupon and a conversion price representing a 40% premium over the stock's closing price at the time of the deal. Concurrent with the transaction, PTC repurchased the majority of its 1.5% senior convertible notes due in September 2026. Cash, cash equivalents, and marketable securities were $2,229.1 million on June 30, 2026, compared to $1,945.4 million on December 31, 2025. Shares issued and outstanding as of June 30, 2026, were 83,327,286. PTC Updates Full-Year 2026 Financial Guidance Expected total revenue increased to $1.18 to $1.28 billion, with total product revenue guidance raised to $850 to $950 million from $750 to $850 million GAAP R&D and SG&A expense guidance remains $775 to $815 million Non-GAAP R&D and SG&A expense guidance remains $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million Non-GAAP Financial Measures In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP R&D and SG&A expense financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in accordance with GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below. Acronyms CHF: Confoederatio Helvetica Francs (Swiss francs) cUHDRS: Composite Unified Huntington's Disease Rating Scale DMD: Duchenne muscular dystrophy FA: Friedreich's ataxia FDA: US Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington's disease NDA: New Drug Application nmDMD: Nonsense mutation Duchenne muscular dystrophy R&D: Research and Development SG&A: Selling, General, and Administrative Today's Conference Call and Webcast Reminder To access the live webcast, please visit the "Events & Presentations" page within the Investors section of the PTC website. A replay of the webcast will be available on the PTC website for 30 days following the event. To participate via phone, please register in advance here to receive dial-in details. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit and follow on LinkedIn, X, Facebook and Instagram. For more information please contact: Investors: Ellen Cavaleri +1 (615) 618-8228 [email protected] Media: Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statements: This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including the information provided under the heading "PTC Updates Full-Year 2026 Financial Guidance", including with respect to (i) 2026 total product revenue guidance and total revenue guidance and (ii) 2026 GAAP and non-GAAP R&D and SG&A expense guidance, and statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, meetings with regulatory agencies, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance," "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," "aim," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to Sephience, including commercialization and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in geographies in which it has been approved and the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna and the withdrawal of the Translarna NDA in the US on other regulatory bodies; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation for votoplam for the treatment of Huntington's disease including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis, the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses, including potential accelerated approval; expectations with respect to Upstaza/Kebilidi, including commercialization, manufacturing capabilities, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to vatiquinone, including with respect to the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses and potential approvals and other matters; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; expectations regarding PTC's product candidates, including the timing of clinical trials and studies; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; PTC's ability to satisfy its obligations under the terms of its lease agreements; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Sephience, Translarna, Emflaza, Upstaza, Kebilidi, Evrysdi, Tegsedi or Waylivra. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. SOURCE PTC Therapeutics, Inc. 21% more press release views with Request a Demo

临床1期财报高管变更临床2期临床3期

2026-08-02

·终身学习-星辰如梦

【药品分享】注射用西罗莫司（白蛋白结合型）——mTOR 激酶抑制剂 || 白蛋白纳米粒递送系统

注射用西罗莫司（白蛋白结合型，ABI-009）依托白蛋白纳米递送平台，为mTOR抑制剂西罗莫司的静脉改良制剂，该剂型核心开发方向为晚期实体瘤，尚处于Ⅱ期临床。基本信息原料药：西罗莫司（雷帕霉素，mTOR抑制剂）制剂技术：白蛋白纳米粒递送系统（与白蛋白紫杉醇Abraxane同平台技术）剂型与规格：注射混悬液，单剂量西林瓶内装有冻干粉末，含100 mg西罗莫司，制备为白蛋白结合型微粒，需复溶后使用。优势对比普通西罗莫司注射液无需使用有毒有机溶剂（普通西罗莫司注射剂含聚氧乙烯蓖麻油，易过敏）；水溶性提升，输注前无需复杂预处理、降低过敏风险；依靠白蛋白受体 gp60/caveolin通路靶向富集于肿瘤组织；静脉给药，适合实体瘤全身给药。口服西罗莫司 vs 白蛋白结合型注射西罗莫司作用机制：西罗莫司为mTOR激酶抑制剂，抑制mTORC1通路，阻断肿瘤细胞增殖、血管生成、代谢重编程，抑制肿瘤相关成纤维细胞、调节肿瘤微环境，同时具有免疫抑制活性。传统西罗莫司主要用于器官移植抗排异；白蛋白结合剂型重点开发抗肿瘤适应症。适应症：西罗莫司是一种 TOR抑制剂，适用于治疗患有局部晚期不可切除或转移性恶性血管周上皮样细胞肿瘤（PEComa）的成人患者。上市情况海外原研（Fyarro，ABI-009，sirolimus protein-bound particles for injectable suspension) (albumin-bound) 注射用西罗莫司蛋白结合混悬液，白蛋白结合型）：2021-11-22 FDA正式获批上市，适应症为成人不可切除局部晚期/转移性恶性PEComa（血管周围上皮样细胞瘤）；目前仅美国获批，尚未在中国进口申报、未在欧盟获批。国内仿制/改良新药（石药HB1901，注射用西罗莫司（白蛋白结合型））国内暂无任何厂家获批上市；石药HB1901(化药2.2类改良新药)用于恶性PEComa的Ib/Ⅲ期研究，2026年4月达到主要终点，持有突破性治疗认定。同时开展上皮样血管内皮瘤等其他适应症Ⅱ期临床。注册与受理信息临床试验信息参比制剂专利情况原研产品专利（制剂/组合物/制备工艺）原研用途专利（适应症/给药方案）中国专利信息石药两条同族专利CN122270278A（中国）与WO2026/067584（PCT）属于同一申请衍生的国内外同族专利。核心改良孵育工艺是在原有石药白蛋白纳米粒平台基础上迭代优化，进一步完善制剂稳定性、临床使用便利性。 SN生物专利保护皮下给药制剂（皮下注射剂型西罗莫司白蛋白纳米粒）。石药HB1901和原研 Fyarro均为静脉输注，其给药途径不同，不存在直接冲突，不会阻碍静脉注射白蛋白结合型西罗莫司开发。鲁南制药专利保护PLGA缓释微球递送体系，PLGA骨架西罗莫司缓释微球注射剂，载体为可降解聚乳酸-羟基乙酸共聚物微球，不属于白蛋白纳米粒体系。与注射用西罗莫司（白蛋白结合纳米粒）制剂技术路线完全不同，不存在专利侵权风险，不会阻碍白蛋白结合型产品的国内研发与申报。其他药品分享链接如下，欢迎点击查看： Ormeloxifene OTF慢性体重管理的新型药物-Tirzepatide新型的非阿片类小分子镇痛药-Suzetrigine奥麦利昔芬口腔薄膜立项调研报告纳米技术双氯芬酸乳胶剂药学研究2025年9月药品批准信息快讯（八）——利斯的明透皮贴剂分享2025年9月药品批准信息快讯（九）——米托坦片分享CDE-《化学仿制药参比制剂目录（第一百批）》（征求意见稿）[附: 药品分享——阿达苏(洛沙平吸入剂)]药品分享-塞来昔布盐酸曲马多片药品分享——伐莫洛龙口服混悬液【药品分享】盐酸菲优拉生片【药品分享】拉坦噻吗滴眼液【药品分享】Baxdrostat 片【药品分享】Omecamtiv Mecarbil缓释片【药品分享】曲地匹坦（Rolapitant）【药品分享】Zasocitinib 胶囊【药品分享】甲磺酸阿帕替尼片【药品分享】巴氯芬口服溶液【药品分享】奥德昔巴特胶囊【药品分享】扎维吉泮鼻喷雾剂【药品分享】左羟丙哌嗪糖浆【药品分享】利丙双卡因凝胶贴膏【药品分享】司来吉兰改良型新药（缓释片）【药品分享】复方甘菊利多卡因凝胶【药品分享】盐酸索安非托片【药品分享】地塞米松口溶膜【药品分享】盐酸来罗西利片【药品分享】马来酸噻吗洛尔凝胶【药品分享】褪黑素颗粒【药品分享】罗氟司特乳膏【药品分享】盐酸芬戈莫德胶囊【药品分享】示踪用盐酸米托蒽醌注射液【药品分享】美洛昔康纳米晶注射液【药品分享】Clascoterone 5%外用溶液【药品分享】马来酸依那普利口服溶液【药品分享】注射用双氯芬酸钠利多卡因【药品分享】马来酸依那普利叶酸片—复方降压药【药品分享】依曲帕米鼻喷雾剂—用于治疗成人阵发性室上性心动过速的可自行给药鼻喷雾剂【药品分享】奥卡西平口服混悬液—一款可用于儿童的钠通道调节类抗癫痫药【药品分享】泊沙康唑口服混悬液——一种常用的抗真菌药物【药品分享】舒沃替尼片【药品分享】依伏卡塞片——第二代口服拟钙剂【药品分享】西诺氨酯片——第三代抗癫痫发作药物【药品分享】匹妥布替尼片——非共价可逆 BTK 抑制剂（用于复发 / 难治套细胞淋巴瘤（MCL））【药品分享】贝美前列素【产品分享】达普司他片【药品分享】水合氯醛糖浆——镇静催眠药【产品分享】苏沃雷生（Suvorexant）——全球首个食欲素受体拮抗剂类催眠药【药品分享】贝沙罗汀【药品分享】地夫可特干混悬剂——一种糖皮质激素类药物【药品分享】伊卢多啉片——治疗成人腹泻型肠易激综合征（IBS‑D）的首创口服阿片受体调节剂【药品分享】卢美哌隆胶囊——一种新型的非典型抗精神病药物【药品分享】奥氟格列隆片——口服非肽类小分子 GLP-1 受体激动剂【药品分享】苯磺酸克利加巴林胶囊——一种新型的治疗神经病理性疼痛的药物【药品分享】苯磺酸美洛加巴林片——治疗周围神经病理性疼痛（PNP）的第三代钙离子通道调节剂【药品分享】Iberdomide胶囊——一款新一代cereblon（CRBN）E3连接酶调节剂（CELMoD）化合物【药品分享】酒石酸伐尼克兰鼻喷雾剂——一种用于治疗干眼症的西药【药品分享】赛沃替尼片——中国首个获批的高选择性 MET 酪氨酸激酶抑制剂【药品分享】盐酸阿夫唑嗪缓释片——一种用于治疗良性前列腺增生（BPH）的 α1 - 受体阻滞剂【药品分享】索格列净片——全球首个获批的SGLT1/SGLT2 双重抑制剂【药品分享】奥吡卡朋胶囊——新一代的儿茶酚-氧位-甲基转移酶抑制剂（COMT）【药品分享】奎扎替尼片——一种口服高选择性 FLT3 抑制剂【药品分享】硫酸拉罗替尼胶囊 / 口服溶液——全球首个高选择性口服 TRK 抑制剂【药品分享】司帕生坦片——成人IgA肾病领域首个非免疫抑制疗法【药品分享】黄体酮阴道栓剂/阴道缓释凝胶剂——主要用于辅助生殖技术（ART）中的黄体支持【药品分享】替戈拉生片——首款国产钾离子竞争性酸阻滞剂类药物（P-CAB）【药品分享】拉坦前列烯酯滴眼剂——一种新型眼科用药【药品分享】布比卡因脂质体注射液——一种长效局部麻醉药【药品分享】甲磺酸加诺沙星片——一种喹诺酮类抗菌药【药品分享】磷酸芦可替尼乳膏——中国首款且唯一获批的白癜风治疗靶向药【药品分享】甲磺酸洛美他派胶囊——全球唯一口服MTP抑制剂【药品分享】依达拉奉口服混悬液——肌萎缩侧索硬化症（ALS，渐冻人症）新药【药品分享】依达拉奉右莰醇舌下片——全球卒中领域首个获FDA突破性疗法认定的舌下脑保护剂【药品分享】马来酸氟诺替尼片——JAK2/FLT3/CDK6 三靶点抑制剂【药品分享】普拉替尼胶囊——国内首个获批的高选择性 RET（转染重排）酪氨酸激酶抑制剂【药品分享】右美托咪定舌下膜——新型 α₂肾上腺素受体激动剂舌下膜剂【药品分享】卡匹色替片——全球首个获批上市的AKT抑制剂【药品分享】库莫西利胶囊——全球首个CDK2/4/6抑制剂【药品分享】赛贝曲司他片——全球首个HAE急性发作口服疗法【药品分享】KYGEVVI（Doxecitine/Doxribtimine）——全球首款且唯一获批的TK2d针对性治疗药物【药品分享】Forzinity（Elamipretide）注射剂——全球首个用于治疗Barth综合征的药物【药品分享】Brinsupri（Brensocatib, 布伦索卡替布）片——全球首个获批上市的二肽基肽酶1（DPP-1）抑制剂【药品分享】维立西呱片——全球首个心衰适应症的可溶性鸟苷酸环化酶（sGC）刺激剂【药品分享】盐酸哌罗匹隆片——非典型抗精神病药【药品分享】盐酸托泊替康胶囊/注射用盐酸托泊替康——经典的拓扑异构酶Ⅰ抑制剂类抗肿瘤药【药品分享】阿普昔腾坦片——全球首个获批用于难治性高血压的口服双重内皮素受体拮抗剂（ERA）【药品分享】注射用盐酸依拉环素——全球首个氟环素类抗生素【药品分享】奥洛格列净胶囊——国内首款抑制SGLT1与SGLT2的1类创新药【药品分享】盐酸匹米替尼胶囊——国内首个、全球首个获批的腱鞘巨细胞瘤（TGCT）系统性靶向药，高选择性CSF-1R抑制剂【药品分享】布地奈德肠溶胶囊——全球首个、中国唯一获批用于原发性IgA肾病对因治疗的靶向药物【药品分享】艾拉莫德片——中国原研、全球首个上市的小分子抗风湿药（csDMARD）【药品分享】索托克拉片——新一代BCL2抑制剂【药品分享】利奈昔巴特片——口服、选择性、可逆性回肠胆汁酸转运体（IBAT/ASBT）抑制剂【药品分享】多替诺雷片——高选择性URAT1抑制剂【药品分享】瑞普泊肽片——口服GLP‑1/GIP双靶点激动剂【药品分享】硫酸索西美雷塞片——口服、强效的KRAS G12C共价抑制剂【药品分享】罗伐昔替尼片——全球首创JAK/ROCK双靶点口服小分子抑制剂【药品分享】埃诺格鲁肽注射液——全球首个获批上市的cAMP偏向型长效GLP-1受体激动剂【药品分享】维培那肽注射液——长效GLP-1受体激动剂【药品分享】普乐司兰钠注射液——全球首个靶向APOC3 mRNA的siRNA降脂药【药品分享】玛仕度肽注射液——全球首个GCG/GLP-1双激动剂【药品分享】瑞米布替尼片——全球首个获批用于CSU的口服靶向药【药品分享】溴莫尼定噻吗洛尔滴眼液——α₂受体激动剂与非选择性 β 受体阻滞剂组成的复方降眼压药【药品分享】吡洛西利片——国内首个CDK2/4/6多靶点抑制剂【药品分享】奥氟格列隆片（Orforglipron） ——中国首个、全球首批上市的口服小分子 GLP-1受体激动剂【药品分享】Copper Histidine注射液（Zycubo）——全球首个、唯一用于治疗儿童门克斯病（Menkes disease）的铜替代注射剂【药品分享】依来格列隆片（Elecoglipron）——口服小分子非肽类GLP-1受体激动剂【药品分享】阿利奈普仑（Aleniglipron）——口服小分子、非肽类、偏向性GLP-1受体激动剂【药品分享】甲磺酸阿美替尼片——中国首个原研三代EGFR酪氨酸激酶抑制剂（TKI）【药品分享】甲磺酸达麦利替尼片——口服高选择性c-MET酪氨酸激酶抑制剂【药品分享】盐酸Acoramidis片——转甲状腺素蛋白（TTR）稳定剂【药品分享】阿莫西林伏诺拉生胃漂浮片——3D打印胃漂浮片【药品分享】盐酸伊可白滞素片——全球首创口服IL‑23受体拮抗剂【药品分享】马来酸吡咯替尼片——中国首个自研的HER1/HER2/HER4不可逆酪氨酸激酶抑制剂（TKI）【药品分享】本维莫德乳膏——全球首创芳香烃受体（AhR）调节剂（TAMA）类皮肤病外用药【药品分享】盐酸莫托咪酯注射液——化药1类静脉麻醉新药【药品分享】注射用罗哌卡因微晶——长效缓释局部麻醉药【药品分享】盐酸伊立替康脂质体注射液——一款针对吉西他滨治疗失败的转移性胰腺癌的二线标准脂质体化疗药【药品分享】甲磺酸氟马替尼片——Bcr-Abl酪氨酸激酶选择性抑制剂【药品分享】注射用阿加糖酶β——国内获批的首个用于治疗法布雷病的药物【药品分享】甲苯磺酸尼拉帕利胶囊——高选择性PARP1/2抑制剂【药品分享】帕米帕利胶囊——强效、选择性PARP1/2抑制剂【药品分享】苯环喹溴铵鼻喷雾剂——选择性M胆碱能受体拮抗剂【药品分享】注射用拉罗尼酶浓溶液——MPS Ⅰ型的特异性酶替代治疗药物【药品分享】海博麦布片——国产自主原研的胆固醇吸收抑制剂【药品分享】盐酸可洛派韦胶囊——直接抗病毒药物（DAA）→ NS5A抑制剂【药品分享】克拉考特酮乳膏——外用、非甾体、选择性雄激素受体（AR）抑制剂【药品分享】非卢替尼片——全球首个用于多发性硬化的口服BTK抑制剂【药品分享】Brenipatide注射液——GLP-1R/GIPR双受体激动剂（继替尔泊肽之后的第二款）【药品分享】罗赛促红素α注射液——国产首个长效重组促红细胞生成素（EPO）创新药【药品分享】贝组替凡片——全球首个口服小分子HIF-2α抑制剂【药品分享】那米司特片——口服选择性PDE4B抑制剂【药品分享】富马酸立康可泮胶囊——一款针对罕见病PNH的口服补体B因子抑制剂【药品分享】醋酸索乐匹尼布片——高选择性脾酪氨酸激酶（Syk）抑制剂【药品分享】甲磺酸艾多替尼片——全球首款专为肺癌脑转移设计的第三代EGFR-TKI 【药品分享】安瑞克芬注射液——全球首个获批镇痛适应症的高选择性外周κ-阿片受体（KOR）激动剂【药品分享】Difelikefalin注射液——全球首个获批的外周选择性κ-阿片受体（KOR）激动剂【药品分享】Obefazimod——口服、靶向 CBC、特异性增强miR-124的首创小分子【药品分享】拉尼兰诺片——口服泛PPAR激动剂【药品分享】舒洛地特注射液——抗血栓药、血管保护剂【药品分享】贝普若韦生注射液——反义寡核苷酸（ASO）类乙肝新药【药品分享】氨酚右敏口服溶液（奥肯能®）——中国内地唯一获批的氨酚右敏口服溶液【药品分享】佩玛贝特片——新型过氧化物酶体增殖物激活受体α/δ双重激动剂 (PPARα/δ) 【药品分享】盐酸依匹斯汀乳膏——外用第二代非镇静抗组胺抗过敏药膏【药品分享】注射用双羟萘酸帕瑞肽微球——一款用于治疗肢端肥大症的第二代长效生长抑素类似物（SRL）微球制剂【药品分享】埃拉菲布拉诺片——治疗原发性胆汁性胆管炎（PBC）的口服靶向新药【药品分享】泽卢克布仑钠注射液——新一代皮下注射型C5补体抑制剂【药品分享】依达拉奉右莰醇注射用浓溶液——一款复方神经保护类注射剂【药品分享】醋酸戈那瑞林注射液——促性腺激素释放激素（GnRH）类药物【药品分享】甲磺酸贝舒地尔片——全球首个选择性ROCK2激酶抑制剂【药品分享】伊托法替布软膏——外用JAK抑制剂（JAK1/JAK3）【药品分享】注射用华卟啉钠——新型肿瘤光动力治疗（PDT）专用光敏剂【药品分享】优替德隆胶囊——口服埃博霉素类微管抑制剂【药品分享】艾普美妥司他片——EZH1/2双靶创新药【药品分享】精氨酸艾曲莫德片——新型选择性鞘氨醇1-磷酸受体（S1P₁）调节剂【药品分享】磷酸芦可替尼乳膏——全球/国内首款、唯一获批用于非节段型白癜风复色的外用JAK1/JAK2选择性抑制剂【药品分享】盐酸右哌甲酯缓释胶囊——治疗6岁及以上注意缺陷多动障碍（ADHD）的一线药物【药品分享】抗菌肽PL-3301口腔凝胶——中国首创的抗菌肽类抗口腔真菌感染药物【药品分享】Zasocitinib (TAK-279) 胶囊——新一代高选择性口服 TYK2 抑制剂【药品分享】注射用Abelacimab——一款全人源IgG1单克隆抗体，靶向凝血因子XI（FXI）【药品分享】Duvakitug注射液——首创性人源化IgG1-λ2单克隆抗体，靶向TL1A（TNFSF15）【药品分享】醋酸氟轻松/氢醌/维A酸乳膏——治疗中重度黄褐斑疗效明确的外用复方【药品分享】富马酸伏诺拉生氯化钠注射液——钾离子竞争性酸阻滞剂（P‑CAB）||东阳光药业研发的2.2,2.4类改良新型新药【药品分享】盐酸阿曲生坦片——诺华研发的高选择性内皮素 A（ETA）受体拮抗剂||中国首个获批用于IgA 肾病的非免疫性口服靶向药【药品分享】RGT-274-CR胶囊——上海齐鲁锐格医药研发的口服小分子GLP-1受体激动剂【药品分享】酒石酸西尼必利片——高选择性5-HT₄受体激动剂类胃肠动力药【药品分享】石杉碱甲控释片——采用双相控释/渗透泵技术的2.2类改良型新药【药品分享】塞伐艾替尼片——口服、可逆、选择性酪氨酸激酶抑制剂（TKI）【药品分享】Admilparant片——口服小分子LPA₁受体拮抗剂【药品分享】瑞拉可兰胶囊——全球首个选择性糖皮质激素受体（GR）拮抗剂【药品分享】盐酸依特卡肽注射液——全球首个、国内唯一获批的静脉注射型拟钙剂【药品分享】Avacincaptad pegol玻璃体内注射液——全球首批用于AMD继发地图样萎缩（GA）的靶向药物【药品分享】盐酸达利雷生片——新一代非管制、非苯二氮䓬类抗失眠药物【药品分享】甲磺酸瑞索利塞片——高选择性PI3Kα小分子靶向抑制剂 || 全球首个获批用于特定卵巢癌的靶向新药【药品分享】富马酸西他沙星注射液——新型喹诺酮类抗菌药物【药品分享】Icotrokinra——全球首个获批的口服 IL-23 受体（IL-23R）选择性拮抗多肽【药品分享】氘可来昔替尼片（Deucravacitinib）——全球首个口服、选择性 TYK2 变构抑制剂【药品分享】柯美奇拜单抗注射液——鼻用糖皮质激素治疗后症状控制不佳的成人中重度季节性过敏性鼻炎患者的福音【药品分享】吉诺昔替尼——口服小分子JAK1/TYK2双重抑制剂【药品分享】洛替拉纳（Lotilaner）——GABA 门控氯离子通道非竞争性拮抗剂 || 兼具兽医驱虫与人用眼科两大用途【药品分享】CagriSema——由卡格列肽（cagrilintide）2.4 mg与司美格鲁肽（semaglutide）2.4 mg组成的强效减重复方药【药品分享】苹果酸卡博替尼——口服、多靶点酪氨酸激酶抑制剂（TKI）【药品分享】迪高替尼（Delgocitinib）——一款外用泛JAK抑制剂【药品分享】拉氟莫司(Laflunimus)——口服免疫抑制剂 || 兼具免疫抑制与抗炎双重活性【药品分享】伏昔尼布片——全球首个IDH1/IDH2双重突变抑制剂靶向药【药品分享】盐酸奥昔布宁片——M受体抗胆碱药【药品分享】贝泽昔替尼片——国内首款获批的高选择性JAK2单靶点抑制剂【药品分享】苯甲酸安达艾替尼胶囊——新型小分子酪氨酸激酶不可逆抑制剂（EGFR-TKI）【药品分享】伏欣奇拜单抗注射液——全人源抗IL‑1β单克隆抗体 || 用于急性痛风性关节炎的1类创新生物药【药品分享】利多卡因丁卡因乳膏【药品分享】伐米奇拜单抗注射液——玻璃体内注射用抗IL-6人源化单抗【药品分享】依赛弗酶α注射液（Efzimfotase alfa 注射液）——第二代重组组织非特异性碱性磷酸酶（TNSALP）酶替代疗法【药品分享】格达托利西布（Gedatolisib）——first‑in-class 泛 PI3K/mTORC1/2 双重抑制剂【药品分享】伯瑞替尼肠溶胶囊——中国原研、高选择性c-MET小分子抑制剂 || 首个获批MET扩增NSCLC单药治疗的靶向药【药品分享】力胜克拉片——中国首个申报上市的原研Bcl-2抑制剂、全球第二款Bcl-2抑制剂【药品分享】BGM0504注射液——GLP-1/GIP 双受体激动剂【药品分享】塞利尼索片——全球首个口服选择性核输出蛋白XPO1抑制剂【药品分享】Seltorexant薄膜包衣片——选择性食欲素2受体（OX2R）高选择性拮抗剂【药品分享】盐酸埃克替尼片——国内首个自主研发的一代EGFR-TKI 【药品分享】盐酸伊普可泮胶囊——全球首个口服补体B因子抑制剂【药品分享】恩考芬尼胶囊——靶向BRAF V600突变的口服激酶抑制剂【药品分享】Enpatoran——口服、选择性TLR7/8小分子抑制剂【药品分享】硝酸异康唑阴道片——抗真菌外用药物【药品分享】Zorevunersen注射液——全球独家ASO上调蛋白表达技术【药品分享】妥拉美替尼胶囊——全球首个针对NRAS突变黑色素瘤的MEK抑制剂【药品分享】盐酸替那帕诺片——全球首创NHE3抑制剂类新型降磷药物【药品分享】注射用莱古比星——新型蒽环类抗肿瘤药【药品分享】优替德隆胶囊——口服埃博霉素类微管抑制剂【药品分享】伊那利塞片——中国首个PIK3CA突变乳腺癌精准靶向药【药品分享】呋喹替尼胶囊——中国首个自主研发VEGFR-TKI || VEGFR靶点靶向抗癌药【药品分享】琥珀酸瑞波西利片——CDK4/6抑制剂【药品分享】盐酸替那帕诺片——全球首个且唯一获批的肠道磷吸收抑制剂【药品分享】芬布替尼片——口服、可逆、可透过血脑屏障的BTK抑制剂【药品分享】炔丙基半胱氨酸胶囊——全球首个进入临床的硫化氢（H₂S）供体I类创新药【药品分享】Balcinrenone达格列净胶囊——选择性盐皮质激素受体拮抗剂（MRA）+SGLT-2抑制剂【药品分享】诺拉西班（Nolasiban）分散片——选择性口服催产素受体拮抗剂 || 早产二线/辅助治疗药物【药品分享】塞多明基注射液——国内首款裸质粒基因治疗药物【药品分享】鲁兹诺雷钠片——国产首个高选择性URAT1抑制剂【药品分享】玛伐凯泰胶囊——全球首创、中国唯一获批的心肌肌球蛋白抑制剂【药品分享】依托芬那酯凝胶——外用非甾体抗炎镇痛药【药品分享】聚卡波非钙片——国际指南首选容积性缓泻剂【药品分享】Mezigdomide胶囊——新一代IMiDs/cereblon E3泛素连接酶调节【药品分享】洛布替尼片——全球首款共价+非共价双机制第四代 BTK 靶向药【药品分享】奥莱泊肽注射液——PEG修饰长效GLP‑1单靶点激动剂【药品分享】安瑞曲替尼胶囊 ——国内第二款国产二代NTRK广谱抗癌药【药品分享】麦格司他胶囊——抑制葡萄糖神经酰胺合成酶的罕见病口服靶向药【药品分享】乌帕替尼片（Upadacitinib）——选择性JAK1抑制剂【药品分享】Dordaviprone胶囊——全球首款、目前唯一获批用于 H3 K27M 突变型弥漫性中线胶质瘤（DMG）的靶向药【药品分享】屈昔多巴胶囊——去甲肾上腺素前体药物 || 帕金森、多系统萎缩、神经源性体位低血压一线对症用药【药品分享】康特替尼颗粒——第二代ALK多靶点激酶抑制剂【药品分享】盐酸兰诺可泮片——国产首个获批上市的补体因子B（CFB）抑制剂【药品分享】Elinzanetant软胶囊——全球首款NK1/NK3双重神经激肽受体拮抗剂【药品分享】Fosmanogepix注射液——全球首创（First-in-class）Gwt1抑制剂类抗真菌前药【药品分享】Zorevunersen注射液——反义寡核苷酸药物【药品分享】宗艾替尼片——高选择性不可逆HER2（ERBB2）酪氨酸激酶抑制剂（TKI）【药品分享】甲苯磺酸他拉唑帕利胶囊——高选择性PARP抑制剂【药品分享】Bexicaserin口服溶液——选择性5-HT2C受体激动剂口服液体制剂【药品分享】醋酸伏诺拉生注射用浓溶液——钾离子竞争性酸阻滞剂（P-CAB）【药品分享】甲苯磺酸依仑司群片——新一代口服选择性雌激素受体降解剂（SERD）【药品分享】硫酸瑞美吉泮口崩片——CGRP 受体拮抗剂（降钙素基因相关肽）|| 偏头痛靶向特效药【药品分享】阿托吉泮片——全球首款口服长效CGRP靶点预防偏头痛药物【药品分享】乌布吉泮片——CGRP受体拮抗剂 || 第二代口服偏头痛急性期药物【药品分享】扎维吉泮鼻喷雾剂——高选择性CGRP受体拮抗剂 || 全球唯一获批鼻喷CGRP吉泮类急性期药物【药品分享】培来加南喷雾剂——全球首款上市加南类多肽抗菌药（FIC）【药品分享】艾沙利酮片——高选择性非甾体醛固酮受体拮抗剂（MRA） || 全球首个上市的非甾体选择性MR拮抗剂【药品分享】布美他尼鼻喷雾剂——全球首款、唯一鼻内给药袢利尿剂【药品分享】注射用盐酸依拉环素——新型全合成氟环素类（四环素衍生物）静脉抗生素【药品分享】舒瑞基奥仑赛注射液——全球首款实体瘤自体CLDN18.2靶向CAR-T细胞新药【药品分享】盐酸环喷托酯滴眼液——儿童验光首选短效睫状肌麻痹剂之一【药品分享】盐酸赛庚啶口服溶液——抗过敏抗组胺药【药品分享】Rinzimetostat (ORIC-944)——口服选择性 EED别构型PRC2表观遗传抑制剂【药品分享】Empasiprubart注射用浓溶液——全球首创靶向补体C2人源化IgG1清扫型单克隆抗体【药品分享】阿更葡糖钠注射液——全球第二款、中国首款自主研发的环糊精类靶向肌松逆转剂【药品分享】盐酸罗哌卡因缓释溶液——国产自主长效局麻缓释制剂 || 填补国内切口原位长效镇痛空白【药品分享】Telisotuzumab Adizutecan 注射用粉末——抗c-Met抗体偶联药物（ADC）【药品分享】L606 (曲前列尼尔脂质体吸入混悬液)——靶向肺动脉高压【药品分享】佩米替尼片——选择性FGFR1/2/3酪氨酸激酶抑制剂【药品分享】唑尼沙胺胶囊——磺酰胺类结构 || 广谱抗癫痫药，偏头痛预防【药品分享】琥珀酸安维吖啶片——双靶点胆碱酯酶抑制剂【药品分享】布莱昔替尼片（Bleximenib）——口服高选择性Menin-KMT2A相互作用抑制剂【药品分享】利厄替尼片——第三代EGFR-TKI靶向药【药品分享】注射用苯磺酸瑞马唑仑——苯二氮䓬类短效静脉镇静/麻醉药【药品分享】瑞维鲁胺片——国产全新二代雄激素受体抑制剂【药品分享】塞普替尼胶囊——全球首款高选择性RET靶向药 || 国内唯一拥有12岁以上儿童甲状腺肿瘤完整临床证据的RET抑制剂【药品分享】注射用和厚朴酚脂质体冻干粉——全球首创小分子脂质体靶向制剂【药品分享】注射用顺铂聚合物胶束——前沿纳米化疗创新赛道 || 改良型新药-纳米靶向注射制剂【药品分享】奥普雷顿片——国内首个获批的口服OX2R选择性激动剂免责声明：本文仅作医药信息传播分享，并不构成投资或决策建议。

100 项与地夫可特相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D03671	地夫可特	H02AB13	DB11921

研发状态

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适应症	国家/地区	公司	日期
杜氏肌营养不良症	意大利	-	1985-01-01

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适应症	最高研发状态	国家/地区	公司	日期
肢带型肌营养不良2I型	临床3期	美国	PTC Therapeutics, Inc.	2019-10-31
肢带型肌营养不良2I型	临床3期	加拿大	PTC Therapeutics, Inc.	2019-10-31
肢带型肌营养不良2I型	临床3期	丹麦	PTC Therapeutics, Inc.	2019-10-31
肢带型肌营养不良2I型	临床3期	法国	PTC Therapeutics, Inc.	2019-10-31
肢带型肌营养不良2I型	临床3期	德国	PTC Therapeutics, Inc.	2019-10-31
肢带型肌营养不良2I型	临床3期	挪威	PTC Therapeutics, Inc.	2019-10-31
肢带型肌营养不良2I型	临床3期	俄罗斯	PTC Therapeutics, Inc.	2019-10-31
肢带型肌营养不良2I型	临床3期	瑞典	PTC Therapeutics, Inc.	2019-10-31
类风湿关节炎	临床1期	中国	-	2013-11-01
类风湿关节炎	临床1期	中国	天津药物研究院药业有限责任公司	2013-11-01

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临床结果

适应症

分期

评价

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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


NCT01603407 (FOR-DMD, CTgov)	临床3期	杜氏肌营养不良症	196	prednisone (Daily Prednisone)	艱淵網夢鏇淵夢遞願遞(顧構糧憲餘淵淵蓋餘遞) = 鑰廠積網鹽醖齋艱網願繭鑰艱糧觸觸淵願構繭 (鬱鹹選築築鏇齋鬱願餘, 0.03) 更多	-	2022-08-12
				Deflazacort (Daily Deflazacort)	艱淵網夢鏇淵夢遞願遞(顧構糧憲餘淵淵蓋餘遞) = 積糧夢壓衊窪鬱壓觸餘繭鑰艱糧觸觸淵願構繭 (鬱鹹選築築鏇齋鬱願餘, 0.03) 更多
NCT03783923 (CTgov)	临床3期	肢带型肌营养不良2I型	11	Deflazacort	繭構簾窪構廠鬱廠廠鑰(選鏇範鹹願選製夢構網) = 鹹顧艱觸鏇蓋構選積艱遞觸壓顧壓鑰鹽衊艱餘 (製齋淵糧積醖鬱餘鹽憲, 2.0178) 更多	-	2022-06-27
AAN2021	N/A	杜氏肌营养不良症	97	Deflazacort 0.45mg/kg/day	構憲憲觸觸簾鑰鬱鑰願(膚構鑰鏇鹹齋窪遞糧淵) = 齋網蓋襯獵壓積夢蓋窪廠構簾襯醖餘獵築淵鏇 (簾積艱顧鏇憲鏇淵餘鬱, 41.5)	不佳	2021-04-13
				Deflazacort 0.9mg/kg/day	構憲憲觸觸簾鑰鬱鑰願(膚構鑰鏇鹹齋窪遞糧淵) = 遞襯製築膚膚獵選醖築廠構簾襯醖餘獵築淵鏇 (簾積艱顧鏇憲鏇淵餘鬱, 44)
AAN2019	临床3期	杜氏肌营养不良症	-	Deflazacort	繭繭構齋廠鹽淵廠觸廠(網壓獵窪艱獵蓋艱觸鹹) = 顧淵艱艱窪鏇範顧網廠膚衊壓網鏇膚襯艱築積 (憲鬱積鏇衊鹹鏇簾鏇鹹, (0.5 ~ 4.1)) 更多	积极	2019-04-09
AAN2018	N/A	杜氏肌营养不良症	-	Deflazacort	艱範鹹淵蓋範遞願壓鬱(夢觸繭鏇積遞壓簾蓋淵) = 餘壓製顧襯鏇淵積積顧鹹窪鑰糧膚鏇製構衊鏇 (構艱構構衊簾選膚遞夢, 13.5)	-	2018-04-10
				Prednisone/Prednisolone	-
CNS2016	临床3期	杜氏肌营养不良症	196	Deflazacort 0.9 mg/kg/day	醖膚製簾鏇簾餘鹹製窪(觸衊餘憲繭獵窪衊憲鏇) = 廠窪簾醖憲淵糧膚膚顧壓衊築鹽願繭繭衊觸衊 (壓網廠襯繭齋獵網獵鹽 )	-	2016-10-14
				Deflazacort 1.2 mg/kg/day	壓廠選窪製衊選鹽鹹鹽(窪製窪膚憲餘構願壓製) = 壓廠選製淵鹹衊鹹餘衊淵壓網艱膚網衊願簾繭 (醖壓積簾廠遞糧積襯鬱 )
AAN 12016 \| AAN 22016	临床3期	杜氏肌营养不良症	196	Deflazacort 0.9 mg/kg/day	範廠觸選選窪觸糧艱遞(鬱選繭膚顧憲築窪選簾) = 淵廠鹹製衊膚壓構鏇獵築窪選選築鬱築鏇選鹽 (衊網廠廠繭積觸廠憲窪 )	积极	2016-04-05
				Deflazacort 1.2 mg/kg/day	觸憲觸醖鬱繭鑰壓夢衊(遞獵窪齋遞齋鏇範壓願) = 廠壓遞鏇齋憲觸憲簾齋構齋壓夢壓鑰鏇繭觸築 (齋鬱鏇顧鹹憲鹽積蓋窪 )