北京若生智能科技有限公司
Drug Database
DA

darbepoetin alfa (Darbatitor)

✓ Approved

Torrent Pharmaceuticals Limited · EPOR · 重组蛋白

什么是 darbepoetin alfa？

darbepoetin alfa 是一种重组蛋白，由Torrent Pharmaceuticals Limited研发。该药已获批，用于治疗相关适应症。

药物档案

商品名Darbatitor
公司Torrent Pharmaceuticals Limited
药物类别重组蛋白
分子靶点EPOR
状态Approved
来源： ClinicalTrials.gov, Torrent Pharmaceuticals Limited

作用机制

分子靶点

darbepoetin alfa 作用于 1 个分子靶点：

EPORerythropoietin receptor (EPO-R)
需要更深入的分析？Noah AI 可解释复杂机制并与同类药物比较。

治疗适应症

darbepoetin alfa 针对 1 个适应症，涉及 1 个治疗领域。

治疗领域疾病/病症分期
Blood and lymphatic system disordersNephrogenic anaemia✓ Approved

相关研究文献

PubMedMolecular genetics and metabolism reports2026-10-27

Olipudase alfa for acid sphingomyelinase deficiency: Evidence of efficacy from a compassionate use program.

Gragnaniello Vincenza V, Tagi Veronica Maria VM, Carubbi Francesca F, Barbato Antonio A et al.

Acid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disorder caused by mutations in the SMPD1 gene, leading to systemic accumulation of sphingomyelin, primarily affecting the liver, spleen, lungs, and central nervous system (CNS). Olipudase alfa represents the first approved enzyme replacement therapy (ERT) targeting the non-neurological manifestations of ASMD. This study evaluates real-world safety and efficacy data of olipudase alfa in a cohort of Italian patients. We retrospectively analyzed clinical, radiological, and laboratory data from 8 Italian patients (5 pediatric, 3 adult) with chronic visceral or chronic neurovisceral ASMD. All patients received olipudase alfa through a compassionate use program between December 2021 and November 2023. Data was collected at baseline, 3, 6, 9, 12, and 15 months of treatment. At baseline, the cohort exhibited hepatosplenomegaly, interstitial lung disease, dyslipidemia, cytopenias, and hypertransaminasemia. Treatment led to significant improvements in hematological parameters, lipid profiles, and liver enzymes. A marked reduction in lyso-sphingomyelin levels was observed, alongside a decrease in spleen and liver volumes. Pulmonary function either improved or remained stable in most patients. While seven patients showed a robust response, one individual exhibited a suboptimal clinical and biochemical clinical outcome. Overall, the therapy was well-tolerated, with a favorable safety profile and few adverse events. In this real-world cohort, olipudase alfa was safe and effective in improving clinical and biochemical markers of ASMD, consistent with results from pivotal clinical trials. However, the observed variability in individual responses emphasizes the importance of personalized monitoring and tailored management strategies.

PMID 42936429
阅读全文 →
PubMedVeterinary sciences2026-10-27

Hematologic Responses to Molidustat in Cats with Anemia of Heterogeneous Etiology: A Retrospective Descriptive Study.

Lee Gippeum G, Lee Jeongmin J, Song Kun-Ho KH

Molidustat, an oral hypoxia-inducible factor prolyl hydroxylase inhibitor, is approved for nonregenerative anemia associated with chronic kidney disease (CKD) in cats but is also used for anemia of other causes. This retrospective, single-center study described the hematologic response to molidustat in 39 cats with anemia of heterogeneous etiology and identified factors that complicate attribution of improvement to the drug. Response was defined as a hematocrit (HCT) increase of ≥4 percentage points from baseline at the complete blood count closest to day 28 (days 14-45), without transfusion to treat anemia. Molidustat was used outside the approved indication in 33 cats (84.6%). Seventeen cats (43.6%) had a response-window measurement; they had a longer treatment span and differed in etiology and concurrent treatment from non-evaluable cats. Among these 17 cats, 12 (70.6%; 95% CI 46.9-86.7%) met the response criterion; sensitivity analyses specified before formal analysis gave 70.6-75.0% and post hoc attribution analyses 29.4-50.0%. In 7 of the 12 responders, darbepoetin or transfusion preceded the qualifying measurement, or it was obtained more than 7 days after the last dose. These data describe clinical use but do not establish effectiveness outside CKD-associated anemia.

PMID 42944170
阅读全文 →
PubMedPharmaceutics2026-10-27

Safety Pharmacology, Toxicokinetics and Pharmacodynamics of VMX-C001, an Intravenous Bypassing Agent for Factor Xa Inhibitors, in Animal Models.

Verhoef Daniël D, Short Gerard G, Gomes Tainá T, Groen Kees K et al.

Background: Anticoagulation therapy confers increased risk of potentially life-threatening bleeding events. Limited options exist for anticoagulation reversal in patients treated with direct oral factor Xa (FXa) anticoagulants (aka FXa inhibitors). Clinical development of reltodecacog alfa (VMX-C001), a modified form of human zymogen FX, as an FXa inhibitor-bypassing agent is ongoing. We report non-clinical pharmacology, toxicokinetic and pharmacodynamic studies of VMX-C001. Methods: Dose-ranging and 2-week toxicity studies were conducted in which Sprague-Dawley rats and cynomolgus monkeys received VMX-C001 at various doses for ≤2 weeks. Safety, pharmacologic, toxicokinetic and pharmacodynamic parameters were assessed. The anticoagulation-bypassing ability of VMX-C001 against edoxaban (ex vivo spiking) and rivaroxaban (in vivo) in monkeys was also evaluated. Results: No safety concerns were noted in either species at VMX-C001 doses up to 10 mg/kg; no VMX-C001-related adverse clinical signs were observed. VMX-C001 exhibited approximately dose-proportional kinetics, with no accumulation after repeat dosing. Changes in thrombin-generation parameters in both species were mild and non-adverse. VMX-C001 restored rivaroxaban-induced decreases in thrombin generation toward pre-rivaroxaban levels. In blood samples spiked ex vivo with edoxaban, the ability of VMX-C001 to support thrombin generation was maintained, even after repeat dosing. Conclusions: Treatment with VMX-C001 for 2 weeks was well tolerated at all tested doses. VMX-C001 had an approximately dose-proportional toxicokinetic profile, and accumulation was not observed upon repeat dosing. VMX-C001 also displayed a favourable pharmacodynamic profile and supported thrombin generation in the presence of FXa inhibitors.

PMID 42942742
阅读全文 →
PubMedPharmaceuticals (Basel, Switzerland)2026-10-27

Multilayered Prioritization of Graph Attention Autoencoder-Derived Drug Repurposing Candidates for COVID-19.

Nakayama Yusuke Y, Tsuji Shingo S, Yamamoto Koji K, Kato-Suzuki Juran J et al.

Background/Objectives: The COVID-19 pandemic highlighted the critical need for drug repurposing. While computational graph-based approaches show promise, single analytical methods often fail to capture the complex pathological mechanisms needed for clinical translation. We aimed to prioritize COVID-19 repurposing candidates by combining computational prediction with orthogonal lines of evidence. Methods: We developed a Graph Attention Autoencoder (GATE) framework with multilayered evaluation integrating computational prediction, real-world data, and phenome analyses. Network-based embeddings identified candidates through latent-space similarity within biomedical knowledge graphs. Clinical relevance was assessed by disproportionality analysis (DPA) of an adverse event reporting system, and biological plausibility through gene expression and pathway profiling. Results: Our GATE framework identified 16 candidate drug entities, including 12 already used or recognized in the context of COVID-19 or its symptoms, consistent with the model recovering known drug-disease associations. The remaining four had minimal prior COVID-19 associations (cilastatin, megestrol, drotrecogin alfa, and ethacrynic acid). Of these four, the multilayered evaluation prioritized two: DPA showed significant inverse reporting associations for COVID-19-related adverse event terms for cilastatin (reporting odds ratio [ROR]: 0.19, 95% confidence interval [CI]: 0.09-0.43) and megestrol (ROR: 0.63, 95% CI: 0.42-0.94). Pathway profiling indicated that cilastatin shares molecular signatures with drugs investigated in COVID-19 clinical trials and megestrol with compounds reported as COVID-19 therapeutic candidates; gene expression analyses suggest cilastatin may exert antiviral and anti-inflammatory effects through DPEP1 inhibition. Conclusions: By integrating GATE-based prediction, real-world evidence, and phenome-level pathway profiling, this study enabled multidimensional candidate prioritization that single analytical methods do not provide. These findings, including the proposed mechanisms of action, are computational predictions that await experimental confirmation. They nonetheless provide mechanistic working hypotheses that may streamline subsequent preclinical studies. Prioritizing clinically approved drugs with established safety profiles may facilitate subsequent clinical evaluation, although the risks specific to each drug would need to be weighed. The framework offers a generalizable strategy for rapid candidate identification during pandemics and diseases with unmet therapeutic needs.

PMID 42943219
阅读全文 →
PubMedSensors (Basel, Switzerland)2026-10-27

Federated Flight Anomaly Detection with Self-Supervised Forecasting: A Flight-Disjoint Evaluation.

Sezgin Anıl A

Reliable anomaly detection is essential for the safe operation of unmanned aerial vehicles (UAVs), particularly in distributed settings where flight telemetry is generated across multiple vehicles or operational sessions and cannot always be centrally collected. This work investigates UAV anomaly detection using models capable of generalizing to unseen flights while keeping raw telemetry within the simulated client partitions during federated training. A self-supervised forecasting framework is evaluated under a simulated federated partitioning of the ALFA corpus and benchmarked using local training, Federated Averaging (FedAvg), FedProx, personalized FedAvg, and centralized learning under a strictly flight-disjoint evaluation protocol. The evaluation comprises 47 complete flights, including 36 failed and 11 nominal flights, grouped into 13 heterogeneous session-based federated clients that serve as statistical partitions rather than physically independent UAVs. The resulting federated setting exhibits non-IID characteristics due to differences in local flight composition and data volume across clients. Federated training is performed for 20 communication rounds with two local epochs per round, and evaluation uses three-fold flight-disjoint cross-validation. Complete flights, including failed flights, are assigned exclusively to either the training or held-out test partition within each fold, ensuring that no samples or overlapping windows from a test flight contribute to model training. An LSTM learns nominal flight dynamics through multivariate next-step forecasting, and the resulting forecasting error is converted into an anomaly score. FedAvg achieves an area under the receiver operating characteristic curve (AUC) of 0.908 and detects 83.3% of failed flights, performing competitively with centralized learning, which obtains an AUC of 0.904 and an 84.4% detection rate. Beyond discrimination performance, calibration, threshold scope, temporal persistence, detection delay, and false-alarm burden are also evaluated. With rolling calibration and a global threshold, the detector maintains an 87.2% flight-level detection rate with a median detection delay of 0.72 s while reducing the false-alarm burden from 202.1 to 66.0 events per hour. However, this alarm frequency remains too high for unattended operational deployment. A more conservative configuration reduces the burden to 8.17 events per hour while retaining 82.8% failed-flight detection and achieving an event precision of 0.851, although the median detection delay increases to 3.38 s. Within this session-partitioned experimental setting, parameter aggregation recovers most of the performance loss associated with local-only training and approaches centralized performance without pooling raw telemetry. These findings should be interpreted as evidence for distributed model aggregation under statistical data partitioning rather than as validation of a fully deployed federated UAV network, while the operational analysis further shows that strong discrimination and flight-level detection do not necessarily translate into practically acceptable alarm behavior.

PMID 42944237
阅读全文 →
PubMedBMJ neurology open2026-10-25

Efficacy and safety of cipaglucosidase alfa plus miglustat in Japanese adults with late-onset Pompe disease: a case series from clinical trials.

Kobayashi Hiroshi H, Narita Aya A, Kawamoto Michi M, Saeki Junjiro J et al.

The safety and efficacy of cipaglucosidase alfa plus miglustat (cipa+mig) versus alglucosidase alfa plus placebo (alg+pbo) in late-onset Pompe disease (LOPD) were established in the randomised, double-blind, 52-week PROPEL study (NCT03729362) conducted across 24 countries, including Japan. An open-label extension (OLE; NCT04138277) is evaluating long-term cipa+mig outcomes. In Japan, cipa+mig was also available before marketing authorisation in an expanded access study (ATB200-19). This case series summarises the efficacy and safety of cipa+mig in Japanese patients in PROPEL, PROPEL OLE and ATB200-19. Endpoints included 6-minute walk distance (6MWD), forced vital capacity (FVC), biomarkers and safety. Cipa+mig (20 mg/kg intravenous cipa and 130-260 mg oral mig (weight-based dosing)) and alg+pbo (20 mg/kg intravenous alg and oral pbo) were given biweekly. Patients randomised to alg+pbo in PROPEL received cipa+mig in the OLE. Safety was assessed at every visit, and biomarkers at weeks 2, 4, 6, 12, 26, 38, 52 and every 26 weeks thereafter. Six patients from PROPEL/OLE (cipa+mig, n=2; alg+pbo, n=4; all enrolled in the OLE) and four from ATB200-19 were included. Two patients discontinued treatment: one in the OLE (anaphylactic reaction) and one in ATB200-19 (withdrawal of consent). For evaluable patients (n=8), 6MWD remained stable or improved from baseline to weeks 52 and 104. In PROPEL/OLE, FVC varied with most patients improving with cipa+mig treatment; in ATB200-19, FVC remained stable to week 104. In PROPEL/OLE and ATB200-19, biomarker levels improved or stabilised by week 104, including in the PROPEL switch group, where levels worsened at week 52 but improved after switching to cipa+mig. Seven patients experienced treatment-related adverse events. No deaths occurred. Although the number of patients was small, the efficacy and safety of cipa+mig in Japanese patients from PROPEL/OLE and ATB200-19 were generally sustained and durable responses consistent with positive results in the overall PROPEL population.

PMID 42921145
阅读全文 →

注册免费账户还可查看另外 9996 篇文献

免费注册查看全部文献 →

了解更多darbepoetin alfa