预约演示
更新于：2026-04-18
Recifercept
更新于：2026-04-18
概要
基本信息
原研机构 |
在研机构- |
非在研机构 |
权益机构- |
最高研发阶段终止临床2期 |
首次获批日期- |
最高研发阶段(中国)终止 |
特殊审评孤儿药 (美国)、孤儿药 (欧盟) |
登录后查看时间轴
结构/序列
Sequence Code 315639340
来源: *****
关联
6
项与 Recifercept 相关的临床试验NCT05061277
A PHASE 1, OPEN-LABEL, SINGLE SUBCUTANEOUS DOSE STUDY TO EVALUATE THE PHARMACOKINETICS, SAFETY AND TOLERABILITY OF LYOPHILIZED FORMULATION OF RECIFERCEPT IN HEALTHY CHINESE PARTICIPANTS
NCT05116046
A PHASE 2 OPEN LABEL EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA
NCT04638153
A PHASE 2 MULTIPLE DOSE, RANDOMIZED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA
100 项与 Recifercept 相关的临床结果
登录后查看更多信息
100 项与 Recifercept 相关的转化医学
登录后查看更多信息
100 项与 Recifercept 相关的专利（医药）
登录后查看更多信息
4
项与 Recifercept 相关的文献（医药）2026-01-01ARCHIVES OF DISEASE IN CHILDHOOD
Sleep-disordered breathing in children with achondroplasia assessed by polysomnography: a retrospective chart review
Article
作者: Adams, Anne-Marie ; Buchwald, Christian von ; Vandeleur, Moya ; Jacobsen, Jeppe Ravn ; Savarirayan, Ravi ; Buciek, Louise Hove ; Raj, Supriya ; Kiaer, Eva Kirkegaard ; Hove, Hanne B ; Griffiths, Amanda
Objectives:
Sleep-disordered breathing is a key childhood complication in children with achondroplasia. This retrospective study aimed to document the prevalence of sleep-disordered breathing in children with achondroplasia assessed by polysomnography.
Design:
The prevalence of sleep-disordered breathing assessed by polysomnography among children aged 0–18 years with achondroplasia from 2013 to 2024 at The Royal Children’s Hospital, Australia, was retrospectively reviewed.
Results:
The cohort included 80 children with achondroplasia (54% females, 95% confirmed molecular diagnosis) with an average number of 3.6 polysomnographies collected per child (n=288). A total of 85% (68/80) had sleep-disordered breathing and 21% reported no prior symptoms. Sleep-disordered breathing subtypes included obstructive sleep apnoea in 81% (55/68), central sleep apnoea in 3% (2/68), mixed sleep apnoea in 7% (5/68) and primary snoring in 9% (6/68). Among those with obstructive and mixed sleep apnoea, 58% (35/60) had moderate or severe obstructive sleep apnoea. In 44 children, a corresponding MRI was evaluated for foramen magnum stenosis using the Achondroplasia Foramen Magnum Score. No correlation was found with sleep-disordered breathing severity (Spearman’s coefficient (ρ)=0.03). Among 27 children who received a precision therapy for achondroplasia (vosoritide, n=18, infigratinib, n=8 and recifercept, n=1), the median respiratory disturbance index/hour improved from 2.7 (25th–75th percentile, (0.9–4.8)) to 1.1 (0.3–2.6) after 1 year of treatment compared with baseline.
Conclusions:
Sleep-disordered breathing was present in 85% of 80 children with achondroplasia, with 21% being asymptomatic. Respiratory parameters did not correlate with foramen magnum stenosis severity and improved after 1 year of treatment in those treated with a precision therapy.
2024-07-01CPT-Pharmacometrics & Systems Pharmacology
Natural disease course modeling of achondroplasia to evaluate the efficacy of recifercept in the absence of a placebo control arm in phase II study
Article
作者: Dawra, Vikas K. ; Qiao, Wenlian ; Boucher, Martin ; Slade, Alison
Abstract:
While randomized, placebo‐controlled, double‐blinded clinical studies are the gold standard for evaluating the efficacy of investigational drugs, the use of placebo in children with achondroplasia should be limited because it provides no clinical benefit while exhausting study participants' treatment window. Recifercept is an investigational drug for treating children with achondroplasia aged 2–10 years. An alternative efficacy evaluation method, instead of a placebo control arm, was employed in the phase II study. Prior to participating in the phase II study, participants completed a natural history (NH) study. Based on the NH data, a multi‐variate linear mixed effects natural disease course model of three anthropometric end points (standing height, sitting height, and arm span) was developed. The model was validated using published growth charts of children with achondroplasia. Subsequently, the model was used to simulate the natural growth trajectories (without any treatment) of the three anthropometric end points for the individuals enrolled in the phase II study. To quantify the efficacy of recifercept, the simulations were compared with the observations post‐recifercept treatment in the phase II study. For all the tested doses of recifercept, at the individual level, the observations were comparable to the simulations at 6 and 12 months post‐recifercept treatment, suggesting no treatment effect. The results contributed to the decision of terminating recifercept clinical development. This work delivers a framework that could eliminate the need for placebo in clinical trials yet provide sufficient evidence for evaluating the efficacy of an investigational drug.
2023-02-01Birth defects research
Juvenile toxicity study of PF ‐07256472/recifercept, a recombinant human soluble fibroblast growth factor receptor 3, in 2–3‐month‐old cynomolgus monkeys
Article
作者: Rana, Payal ; Cappon, Gregg D. ; Fuchs, Antje ; Campion, Sarah N. ; Karanian, David ; Bowman, Christopher J.
Abstract:
Achondroplasia is an autosomal disorder caused by point mutation in the gene encoding fibroblast growth factor receptor 3 (FGFR3) and resulting in gain of function. Recifercept is a potential disease modifying treatment for achondroplasia and functions as a decoy protein that competes for ligands of the mutated FGFR3. Recifercept is intended to restore normal bone growth by preventing the mutated FGFR3 from negative inhibitory signaling in pediatric patients with achondroplasia. Here we evaluated the potential effects of twice weekly administration of recifercept to juvenile cynomolgus monkeys (approximately 3‐months of age at the initiation of dosing) for 6‐months. No adverse effects were noted in this study, identifying the high dose as the no‐observed‐adverse‐effect‐level and supporting the use of recifercept in pediatric patients from birth. Considering that juvenile toxicity studies in nonhuman primates are not frequently conducted, and when they are conducted they typically utilize animals ≥9 months of age, this study demonstrates the feasibility of executing a juvenile toxicity study in very young monkeys prior to weaning.
100 项与 Recifercept 相关的药物交易
登录后查看更多信息
研发状态
10 条进展最快的记录, 后查看更多信息
登录
| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 软骨发育不全 | 临床2期 | 美国 | 2020-12-02 | |
| 软骨发育不全 | 临床2期 | 日本 | 2020-12-02 | |
| 软骨发育不全 | 临床2期 | 澳大利亚 | 2020-12-02 | |
| 软骨发育不全 | 临床2期 | 比利时 | 2020-12-02 | |
| 软骨发育不全 | 临床2期 | 丹麦 | 2020-12-02 | |
| 软骨发育不全 | 临床2期 | 意大利 | 2020-12-02 | |
| 软骨发育不全 | 临床2期 | 葡萄牙 | 2020-12-02 | |
| 软骨发育不全 | 临床2期 | 西班牙 | 2020-12-02 |
登录后查看更多信息
临床结果
临床结果
适应症
分期
评价
查看全部结果
临床2期 | 60 | (Recifercept 1 mg/kg QW) | 範顧淵築壓遞簾壓觸膚 = 壓鏇鏇糧築醖鹽蓋願膚 選範範衊餘艱糧糧築繭 (艱鬱糧餘構夢鹹範製觸, 糧窪糧積製艱夢壓餘遞 ~ 窪夢艱鑰醖壓淵觸鏇壓) 更多 | - | 2024-02-15 | ||
(Recifercept 2 mg/kg BIW) | 範顧淵築壓遞簾壓觸膚 = 鑰衊餘齋繭艱淵獵簾網 選範範衊餘艱糧糧築繭 (艱鬱糧餘構夢鹹範製觸, 積積簾製願艱網憲餘網 ~ 醖壓鹹願蓋鹽窪鏇衊夢) 更多 | ||||||
临床2期 | 35 | (Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly) | 壓餘獵鏇簾艱醖構糧夢 = 鑰構夢構壓鹽網遞製觸 衊艱鬱顧獵鑰齋鏇遞襯 (膚構築選鏇積鹽願簾蓋, 廠膚顧築糧範壓簾願鏇 ~ 襯範觸積願選製醖積餘) 更多 | - | 2024-02-08 | ||
(Recifercept 2 mg/kg Twice Weekly) | 壓餘獵鏇簾艱醖構糧夢 = 鹽遞鏇窪鑰鹽鬱鬱鹹糧 衊艱鬱顧獵鑰齋鏇遞襯 (膚構築選鏇積鹽願簾蓋, 糧繭顧築齋鹽鬱淵窪鹹 ~ 餘鹽鹽齋遞夢製廠選餘) 更多 |
登录后查看更多信息
转化医学
使用我们的转化医学数据加速您的研究。
登录
或
药物交易
使用我们的药物交易数据加速您的研究。
登录
或
核心专利
使用我们的核心专利数据促进您的研究。
登录
或
临床分析
紧跟全球注册中心的最新临床试验。
登录
或
批准
利用最新的监管批准信息加速您的研究。
登录
或
生物类似药
生物类似药在不同国家/地区的竞争态势。请注意临床1/2期并入临床2期，临床2/3期并入临床3期
登录
或
特殊审评
只需点击几下即可了解关键药物信息。
登录
或
生物医药百科问答
全新生物医药AI Agent 覆盖科研全链路，让突破性发现快人一步
立即开始免费试用!
智慧芽新药情报库是智慧芽专为生命科学人士构建的基于AI的创新药情报平台，助您全方位提升您的研发与决策效率。
立即开始数据试用!
智慧芽新药库数据也通过智慧芽数据服务平台，以API或者数据包形式对外开放，助您更加充分利用智慧芽新药情报信息。
生物序列数据库
生物药研发创新
免费使用
化学结构数据库
小分子化药研发创新
免费使用